Abel, M.

Short-Term Effects of Selective Dorsal Rhizotomy on Gait in Children with Bilateral Spastic Cerebral Palsy

Abicht, A.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Liquid Biopsy: Short-Cut to Identify Mosaic Causes of Hemihypertrophic Overgrowth

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Aeschimann-Huhn, R.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Afzal, E.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Ahrens-Nicklas, R. C.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Aiuti, A.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Alam, C. Al

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Alawi, M.

INPP4A-Related Genetic and Phenotypic Spectrum and Functional Relevance of Subcellular Targeting of INPP4A Isoforms

Alber, M.

Homozygous Frameshift Mutation in the ATP1A2 Gene Leading to Severe Pseudo-TORCH Syndrome

Benign Tonic Downgaze of Infancy (Pseudo-Sunset Phenomenon)

Alecu, I.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Aledo-Serrano, Á.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

AlShamsi, A.

Biallelic Variants in OAS2 with Neurodevelopmental Disorders, Skeletal Dysplasia, and Immunodeficiency

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Altmüller, J.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Alvi, J. R.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Amar, R.

Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1)

Amin, S.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Antebi, A.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Biallelic Variants in OAS2 with Neurodevelopmental Disorders, Skeletal Dysplasia, and Immunodeficiency

Arce, V. San Antonio

Neocortical and Mesial Temporal Sleep Spindles Are Reduced in Children with Focal Epilepsy and Developmental Delay

Armangue, T.

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Armangué, T.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Arzimanoglou, A.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Avigdor, T.

Pulsatile Dexamethasone Therapy Reduces Epileptic Burden and Improves Sleep Physiology in Children with Genetic Drug-Resistant Epilepsy

Azam, M.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Bae-Gartz, I.

Expanding Clinical and Genetic Spectrum of IL-6 Signal Transduction Disorders Reveal Variable Immunodeficiency and Neurodevelopmental Disorders with Dysregulated Vesicular Trafficking

Bager, P.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Bakhtiari, S.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Bakker, D. P.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Baldoli, C.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Ballabio, A.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Baranello, G.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Barseghyan, H.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Bartels, F.

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Influence of Fingolimod Treatment on Disease Outcome and MRI Brain Volumes in Children with CLN 3

Baud, M.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Bauder, F.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Baum, E.

LYST-Associated Chediak-Higashi Syndrome: Systematic Review and Reanalysis of Neurological Disorders in 130 Patients

Baumann, B. Tro

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Baumann, M.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Baur, F.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Biallelic Variants in OAS2 with Neurodevelopmental Disorders, Skeletal Dysplasia, and Immunodeficiency

Beblo, S.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Becker, L.-L.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Behne, R.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Behrendt, P.

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

Beiner, M.

Diagnostic and Therapeutic Challenges in a Child with Suspected Hemiplegic Migraine

Bendszus, M.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Benet-Pages, A.

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Ben-Omran, T.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Bernardo, R.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Bernert, G.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Bertolini, A.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Treatment Response in Children with Relapsing MOGAD

Influence of Fingolimod Treatment on Disease Outcome and MRI Brain Volumes in Children with CLN 3

Berulava, T.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Berweck, S.

Short-Term Effects of Selective Dorsal Rhizotomy on Gait in Children with Bilateral Spastic Cerebral Palsy

Sequential Seizures in Neonates: Videos as a Diagnostic Tool for Diagnosis of KCNQ2-Related Self-Limiting Familial Neonatal Epilepsy?

Bettica, P.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Bettini, E.

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Biebl, A.

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Bigi, S.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Biswas, S.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Bittigau, P.

Weathering the Storm: Early Intervention for GAD65 Antibody-Mediated Encephalitis with Drug-Resistant Autoimmune Epilepsy

Blaschek, A.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Treatment Response in Children with Relapsing MOGAD

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Bley, A.

Description of the Hamburg Alexander Leukodystrophy Cohort

Böckenholt, K.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Bondade, S.

Cerliponase Alfa for the Treatment of CLN2 Disease in a Patient Cohort Including Children under 3 Years

Bonfert, M.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Borggräfe, I.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

Borgmann, I.

Roifman Syndrome Is a Rare but Important Differential Diagnosis in Patients Suspected to Have CDG Syndrome

Börner, C.

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

Bramlage, P.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Brantner-Inthaler, S.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Braun, D.

Diagnostic and Therapeutic Challenges in a Child with Suspected Hemiplegic Migraine

Brechbühl, D.

Diagnostic and Therapeutic Challenges in a Child with Suspected Hemiplegic Migraine

Brecht, I.

Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1)

Breu, M.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Brilot, F.

Treatment Response in Children with Relapsing MOGAD

Brogna, C.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Brooks, J.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Brunetti-Pierri, N.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Bültmann, E.

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Busemann, E.-M.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Cadieux-Dion, M.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Cairns, A.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Calabrese, P.

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Calbi, V.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Cardellini, L.

Weathering the Storm: Early Intervention for GAD65 Antibody-Mediated Encephalitis with Drug-Resistant Autoimmune Epilepsy

Cazzaniga, S.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Ceulemans, B.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Chanson, C.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Chen, J. J.

Treatment Response in Children with Relapsing MOGAD

Chen, S.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Chi, A. Nguyen

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Chien, Y.-H.

Eladocagene Exuparvovec Gene Therapy Increases Bayley-III Cognitive and Language Raw Scores in Patients with Aromatic ʟ-Amino Acid Decarboxylase Deficiency

Chitnis, T.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Chou, C.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Christ, J.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Christen, H.-J.

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

Chung, J.

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Cirak, S.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Classen, G.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Cleaveland, R.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Cohen-Pfeffer, J.

Cerliponase Alfa for the Treatment of CLN2 Disease in a Patient Cohort Including Children under 3 Years

Comi, G.

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Conrad, C.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Cooper, G. M.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Cross, J. H.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Dafsari, H.

Expanding Clinical and Genetic Spectrum of IL-6 Signal Transduction Disorders Reveal Variable Immunodeficiency and Neurodevelopmental Disorders with Dysregulated Vesicular Trafficking

Dafsari, H. S.

LYST-Associated Chediak-Higashi Syndrome: Systematic Review and Reanalysis of Neurological Disorders in 130 Patients

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Biallelic Variants in OAS2 with Neurodevelopmental Disorders, Skeletal Dysplasia, and Immunodeficiency

Expanding Clinical and Genetic Spectrum of IL-6 Signal Transduction Disorders Reveal Variable Immunodeficiency and Neurodevelopmental Disorders with Dysregulated Vesicular Trafficking

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Dahl, S. vom

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Dale, R.

Treatment Response in Children with Relapsing MOGAD

Dargvainiene, J.

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

Deiva, K.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Treatment Response in Children with Relapsing MOGAD

Denecke, J.

INPP4A-Related Genetic and Phenotypic Spectrum and Functional Relevance of Subcellular Targeting of INPP4A Isoforms

Derichs, U.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Devinsky, O.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Dierks, T.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Dill, P.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Dobrinsky, C.

Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1)

Doll, T.

A Deceptive Bump: The Case of a Complicated Pott's Puffy Tumor in a Symptom-Free Child

Dreha-Kulaczewski, S.

Regional White Matter Hypertrophy Follows Cerebral ABCD1 Gene Expression Pattern in Asymptomatic X-Linked Adrenoleukodystrophy

Drenckhahn, A.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

NfL as a Biomarker in Monitoring Pediatric MS Patients

Driever, P. Hernáiz

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Drube, J.

Therapeutic Apheresis in Pediatric Patients with Acute Neurological Disorders

Ebrahimi-Fakhari, D.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Eckenweiler, M.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Ocular Myasthenia: It's Worth a Second Look

Efthymiou, S.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Eisenkölbl, A.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Eisenreich, D.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Erdmann, H.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Essing, M.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Evans, A.

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Evans, R.

Long-Term Comparative Efficacy and Safety of Risdiplam versus Nusinersen in Children with Type 1 Spinal Muscular Atrophy (SMA)

Ezgü, F.

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Fabri, N.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Farrar, M.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Fellmeth, R. H.

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

Finke, C.

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Finsterwalder, J.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Fischer, A.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Fitzner, D.

Region-Specific Lipidomic Profiling in Brain and Spinal Cord Tissue of X-Linked Adrenoleukodystrophy Mice

Flotats-Bastardas, M.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Foiadelli, T.

Treatment Response in Children with Relapsing MOGAD

Forbes, S.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Franz, A.

Homozygous Frameshift Mutation in the ATP1A2 Gene Leading to Severe Pseudo-TORCH Syndrome

Frauscher, B.

Pulsatile Dexamethasone Therapy Reduces Epileptic Burden and Improves Sleep Physiology in Children with Genetic Drug-Resistant Epilepsy

Freischläger, F.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Freisinger, P.

Type 1 Interferon Activation in Mitochondrial Disease: A Possible “New” Pathomechanism

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

French, D. L.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Friedrich, S.

Ocular Myasthenia: It's Worth a Second Look

Friese, J.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Froeba-Pohl, A.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Fröhlich, C.

Type 1 Interferon Activation in Mitochondrial Disease: A Possible “New” Pathomechanism

Fucik, P.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Fumagalli, F.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Gagne, A.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Galiart, E.

Impact of Preventable Respiratory Tract Infections on Spinal Muscular Atrophy with Focus on Respiratory Syncytial Virus Infections: A Retrospective Single-Center Study

Gallo, V.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Gammenthaler, C.

Diagnostic and Therapeutic Challenges in a Child with Suspected Hemiplegic Migraine

Garbade, S. F.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

García, A. Mendoza

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Garcia-Tarodo, S.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Gärtner, J.

Region-Specific Lipidomic Profiling in Brain and Spinal Cord Tissue of X-Linked Adrenoleukodystrophy Mice

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Regional White Matter Hypertrophy Follows Cerebral ABCD1 Gene Expression Pattern in Asymptomatic X-Linked Adrenoleukodystrophy

Gastaldi, M.

Ocular Myasthenia: It's Worth a Second Look

Gehrckens, A.

LYST-Associated Chediak-Higashi Syndrome: Systematic Review and Reanalysis of Neurological Disorders in 130 Patients

Geis, T.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Gelb, M. H.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Gellrich, J.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Gerstl, L.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Gil-Nagel, A.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Giorgi, L.

Treatment Response in Children with Relapsing MOGAD

Gissen, P.

Cerliponase Alfa for the Treatment of CLN2 Disease in a Patient Cohort Including Children under 3 Years

Giugliani, R.

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Gkalimani, I.

Regional White Matter Hypertrophy Follows Cerebral ABCD1 Gene Expression Pattern in Asymptomatic X-Linked Adrenoleukodystrophy

Glatter, S.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Gleeson, J.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Gleich, F.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Goeggel-Simonetti, B.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Goemans, N.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Goj, G.

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Influence of Fingolimod Treatment on Disease Outcome and MRI Brain Volumes in Children with CLN 3

Goldhahn, K.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Gordon, G.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Graf, T.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Grasshoff, U.

Homozygous Frameshift Mutation in the ATP1A2 Gene Leading to Severe Pseudo-TORCH Syndrome

Gravesande, K. Storm van's

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Groeschel, S.

Benign Tonic Downgaze of Infancy (Pseudo-Sunset Phenomenon)

Grohmann-Held, K.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Gröschel, S.

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Grünert, J.

Eladocagene Exuparvovec Gene Therapy Increases Bayley-III Cognitive and Language Raw Scores in Patients with Aromatic ʟ-Amino Acid Decarboxylase Deficiency

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Grünert, S. C.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Gschaidmeier, A.

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Long-Term Negative Impact of Pediatric Stroke on Language

Gulieva, S.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Guliyeva, U.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Haack, T.

Homozygous Frameshift Mutation in the ATP1A2 Gene Leading to Severe Pseudo-TORCH Syndrome

Haas, D.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Haas, S.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Haase, C.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Haas-Lude, K.

Benign Tonic Downgaze of Infancy (Pseudo-Sunset Phenomenon)

Haberl, H.

Short-Term Effects of Selective Dorsal Rhizotomy on Gait in Children with Bilateral Spastic Cerebral Palsy

Hackenberg, A.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Hagedorn, C.

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Hahn, A.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Hallermayr, A.

Liquid Biopsy: Short-Cut to Identify Mosaic Causes of Hemihypertrophic Overgrowth

Harder, L.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Harms, F. L.

INPP4A-Related Genetic and Phenotypic Spectrum and Functional Relevance of Subcellular Targeting of INPP4A Isoforms

Hartmann, H.

Therapeutic Apheresis in Pediatric Patients with Acute Neurological Disorders

Hashemi-Gorji, F.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Hasselmann, O.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Häusler, M.

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Häusler, M. G.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Hawkins, N.

Long-Term Comparative Efficacy and Safety of Risdiplam versus Nusinersen in Children with Type 1 Spinal Muscular Atrophy (SMA)

Hecher, L.

INPP4A-Related Genetic and Phenotypic Spectrum and Functional Relevance of Subcellular Targeting of INPP4A Isoforms

Heese, B.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Heiland, S.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Heimgärtner, M.

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Long-Term Negative Impact of Pediatric Stroke on Language

Heinen, F.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Heinrich, K.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Helfferich, J.

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Hengstler, J.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Hennermann, J. B.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Hentrich, L.

Biallelic Variants in OAS2 with Neurodevelopmental Disorders, Skeletal Dysplasia, and Immunodeficiency

Herbst, Z. M.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Hernáiz-Driever, P.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Hernàndez, H. Salvador

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Hiatt, S. M.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Hillmann, S.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Hiz, S.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Hoffmann, G. F.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Hoffmann, J.

Roifman Syndrome Is a Rare but Important Differential Diagnosis in Patients Suspected to Have CDG Syndrome

Höfinger, M.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Hofmann, M.

Elevated TNF-α Levels and CD4 Cell Counts in the Blood of Children with Tuberous Sclerosis Complex (TSC)-Related Refractory Epilepsy

Hofstetter, P.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Holinski-Feder, E.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Holler, A.-S.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Hoopmann, M.

Homozygous Frameshift Mutation in the ATP1A2 Gene Leading to Severe Pseudo-TORCH Syndrome

Hörbe-Blindt, A.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Horber, V.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Horvath, R.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Hösl, M.

Short-Term Effects of Selective Dorsal Rhizotomy on Gait in Children with Bilateral Spastic Cerebral Palsy

Houlden, H.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Huda, S.

Treatment Response in Children with Relapsing MOGAD

Hulliger, L.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Huppke, P.

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Husain, R. A.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Hwu, P. W.-L.

Eladocagene Exuparvovec Gene Therapy Increases Bayley-III Cognitive and Language Raw Scores in Patients with Aromatic ʟ-Amino Acid Decarboxylase Deficiency

Ibadova, R.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Iftikhar, K.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Illsinger, S.

Therapeutic Apheresis in Pediatric Patients with Acute Neurological Disorders

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Ilyas, M.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Imdad, M.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Jacobs, J.

Neocortical and Mesial Temporal Sleep Spindles Are Reduced in Children with Focal Epilepsy and Developmental Delay

Jain, V.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Jaiser, M.

Neocortical and Mesial Temporal Sleep Spindles Are Reduced in Children with Focal Epilepsy and Developmental Delay

Janzen, N.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Jende, J.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Johannsen, J.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Johnsen, C.

Roifman Syndrome Is a Rare but Important Differential Diagnosis in Patients Suspected to Have CDG Syndrome

Jong, Y.-J.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Jóźwiak, S.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Jung, E.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

Jung, E. L.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Jung, S.

Elevated TNF-α Levels and CD4 Cell Counts in the Blood of Children with Tuberous Sclerosis Complex (TSC)-Related Refractory Epilepsy

Jungbluth, H.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Kaindl, A. M.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Weathering the Storm: Early Intervention for GAD65 Antibody-Mediated Encephalitis with Drug-Resistant Autoimmune Epilepsy

A Deceptive Bump: The Case of a Complicated Pott's Puffy Tumor in a Symptom-Free Child

Kaiyrzhanov, R.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Kalbe, E.

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Kalser, J.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Kälviäinen, R.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Kamrath, C.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Kan, H. E.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Kandler, I.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Kanzelmeyer, N.

Therapeutic Apheresis in Pediatric Patients with Acute Neurological Disorders

Karachunski, P.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Karagoz, I.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Karenfort, M.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Treatment Response in Children with Relapsing MOGAD

Karimiani, E. G.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Karydi, A.

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Kasper, D.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Kauffmann, B.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Kaur, A.

Biallelic Variants in OAS2 with Neurodevelopmental Disorders, Skeletal Dysplasia, and Immunodeficiency

Kauth, F.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Kessler, J.

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Kettwig, M.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Roifman Syndrome Is a Rare but Important Differential Diagnosis in Patients Suspected to Have CDG Syndrome

Kirschner, J.

Ocular Myasthenia: It's Worth a Second Look

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Kiwull, L.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Kläger, H.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Klein, A.

Diagnostic and Therapeutic Challenges in a Child with Suspected Hemiplegic Migraine

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Klein, M.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Kleinle, S.

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Klotz, K. A.

Neocortical and Mesial Temporal Sleep Spindles Are Reduced in Children with Focal Epilepsy and Developmental Delay

Kluger, G.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Sequential Seizures in Neonates: Videos as a Diagnostic Tool for Diagnosis of KCNQ2-Related Self-Limiting Familial Neonatal Epilepsy?

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Knierim, E.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Weathering the Storm: Early Intervention for GAD65 Antibody-Mediated Encephalitis with Drug-Resistant Autoimmune Epilepsy

Koch, A.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Koehler, U.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Köhler, C.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Kokaliaris, C.

Long-Term Comparative Efficacy and Safety of Risdiplam versus Nusinersen in Children with Type 1 Spinal Muscular Atrophy (SMA)

Kokaly, N.

Description of the Hamburg Alexander Leukodystrophy Cohort

Kölker, S.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Kolorz, P.

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

Korenke, G.-C.

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

Kornek, B.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Kortas, A.

Primary Headache Types and Quality of Life in Children with Epilepsy

Pulsatile Dexamethasone Therapy Reduces Epileptic Burden and Improves Sleep Physiology in Children with Genetic Drug-Resistant Epilepsy

Sleep Macro- and Microstructure Is Altered in Children with Epilepsy

Koukou, G.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Kousoulos, L.

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

Kreiter, B.

Diagnostic and Therapeutic Challenges in a Child with Suspected Hemiplegic Migraine

Kreppel, F.

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Kreye, J.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Kruer, M.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Kudernatsch, M.

Short-Term Effects of Selective Dorsal Rhizotomy on Gait in Children with Bilateral Spastic Cerebral Palsy

Kuehni, C.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Kuhlmann, P. H.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

Kunstmann, E.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Kurz, F.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Kusnik, S.

Elevated TNF-α Levels and CD4 Cell Counts in the Blood of Children with Tuberous Sclerosis Complex (TSC)-Related Refractory Epilepsy

Kutsche, K.

INPP4A-Related Genetic and Phenotypic Spectrum and Functional Relevance of Subcellular Targeting of INPP4A Isoforms

Lachhammer, T.

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

Lagae, L.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Lai, F.

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Lamersdorf, C.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Lang, P.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Lattau, S. S. J.

Region-Specific Lipidomic Profiling in Brain and Spinal Cord Tissue of X-Linked Adrenoleukodystrophy Mice

Laugwitz, L.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Lauseker, M.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Learoyd, M.

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Lechner, C.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Lee, N.-C.

Eladocagene Exuparvovec Gene Therapy Increases Bayley-III Cognitive and Language Raw Scores in Patients with Aromatic ʟ-Amino Acid Decarboxylase Deficiency

Lee-Kirsch, M. A.

Type 1 Interferon Activation in Mitochondrial Disease: A Possible “New” Pathomechanism

Leiz, S.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Leppmeier, V.

Sequential Seizures in Neonates: Videos as a Diagnostic Tool for Diagnosis of KCNQ2-Related Self-Limiting Familial Neonatal Epilepsy?

Leuzzi, V.

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Leypoldt, D.

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

Lidzba, K.

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Long-Term Negative Impact of Pediatric Stroke on Language

Lieftüchter, V.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Lin, M.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Lindner, M.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Lindt, E.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Lisfeld, J.

INPP4A-Related Genetic and Phenotypic Spectrum and Functional Relevance of Subcellular Targeting of INPP4A Isoforms

Lochmüller, H.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Ocular Myasthenia: It's Worth a Second Look

Löhr-Nilles, C.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Lokhande, H.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

López-Cabeza, S.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Lorentzos, M.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Lothe, A.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Lupo, P.

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Mader, S.

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Maenner, G.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Maguire, J. A.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Mah, J. K.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Mahajan, A.

Long-Term Comparative Efficacy and Safety of Risdiplam versus Nusinersen in Children with Type 1 Spinal Muscular Atrophy (SMA)

Maier, E. M.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Maier, O.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Makridis, K.

Weathering the Storm: Early Intervention for GAD65 Antibody-Mediated Encephalitis with Drug-Resistant Autoimmune Epilepsy

Mall, V.

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Mammadova, D.

Elevated TNF-α Levels and CD4 Cell Counts in the Blood of Children with Tuberous Sclerosis Complex (TSC)-Related Refractory Epilepsy

Mansilla, D.

Pulsatile Dexamethasone Therapy Reduces Epileptic Burden and Improves Sleep Physiology in Children with Genetic Drug-Resistant Epilepsy

Maqbool, S.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Marina, A. Della

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Mariotto, S.

Treatment Response in Children with Relapsing MOGAD

Maroofian, R.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Marquardt, I.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Marquardt, T.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Marsch, B.

Influence of Fingolimod Treatment on Disease Outcome and MRI Brain Volumes in Children with CLN 3

Marten, L. M.

Region-Specific Lipidomic Profiling in Brain and Spinal Cord Tissue of X-Linked Adrenoleukodystrophy Mice

Martin, L.

Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1)

A Deceptive Bump: The Case of a Complicated Pott's Puffy Tumor in a Symptom-Free Child

Marx, M.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Matysiak, K.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Mayer, K.

Efficient Molecular Genetic Diagnostics in Tuberous Sclerosis Complex (TSC) by Deep Next-Generation Sequencing (NGS) of the Entire Genomic TSC Gene Regions Discovers Challenging Variants

McDonald, C.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

McDonald, C. M.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

McMillan, H.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Meier, K.

Regional White Matter Hypertrophy Follows Cerebral ABCD1 Gene Expression Pattern in Asymptomatic X-Linked Adrenoleukodystrophy

Meinl, E.

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Mercuri, E.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Merkenschlager, A.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Mindermann, C.

Therapeutic Apheresis in Pediatric Patients with Acute Neurological Disorders

Miryounesi, M.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Moiseenko, A.

Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1)

Monazahian, M.

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

Monfregola, J.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Mori, A. Capone

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Muensterer, O.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Müller-Felber, W.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Munell, F.

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Munk, I.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Muntoni, F.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Mütze, U.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Møller, R.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Nabbout, R.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Nägele, T.

Homozygous Frameshift Mutation in the ATP1A2 Gene Leading to Severe Pseudo-TORCH Syndrome

Naggar, I. El

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Nam, J.

Long-Term Comparative Efficacy and Safety of Risdiplam versus Nusinersen in Children with Type 1 Spinal Muscular Atrophy (SMA)

Nemeth, G. Oesch

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Nessler, S.

Region-Specific Lipidomic Profiling in Brain and Spinal Cord Tissue of X-Linked Adrenoleukodystrophy Mice

Neu, A.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Neugebauer, J.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Neuhann, T.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Liquid Biopsy: Short-Cut to Identify Mosaic Causes of Hemihypertrophic Overgrowth

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Nikolaus, M.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

NfL as a Biomarker in Monitoring Pediatric MS Patients

A Deceptive Bump: The Case of a Complicated Pott's Puffy Tumor in a Symptom-Free Child

Nikolaus, M. J.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Weathering the Storm: Early Intervention for GAD65 Antibody-Mediated Encephalitis with Drug-Resistant Autoimmune Epilepsy

Niks, E.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Nosadini, M.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Noßwitz, U.

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Obradovic, V.

Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1)

Ojeda, N. Meave

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Olive, G.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Olivieri, M.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

Osman, K.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Ottenberger, A.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Otto, M.

Influence of Fingolimod Treatment on Disease Outcome and MRI Brain Volumes in Children with CLN 3

Palm, K.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Panagi, M.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Panzer, A.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Influence of Fingolimod Treatment on Disease Outcome and MRI Brain Volumes in Children with CLN 3

Paolini, M.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

Partridge, C.-A.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Passos, J.

Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1)

Pearl, P. L.

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Pechmann, A.

Ocular Myasthenia: It's Worth a Second Look

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Pena, T.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Penematsa, V.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Perret-Hoigné, E.

Diagnostic and Therapeutic Challenges in a Child with Suspected Hemiplegic Migraine

Peterson, L.

Efficient Molecular Genetic Diagnostics in Tuberous Sclerosis Complex (TSC) by Deep Next-Generation Sequencing (NGS) of the Entire Genomic TSC Gene Regions Discovers Challenging Variants

Pfeifer, T.

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

Plecko, B.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Polega, S.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Poppel, J.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Pospieszny, K.

Diagnostic and Therapeutic Challenges in a Child with Suspected Hemiplegic Migraine

Potratz, C.

Weathering the Storm: Early Intervention for GAD65 Antibody-Mediated Encephalitis with Drug-Resistant Autoimmune Epilepsy

Preisel, M.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Preisner, F.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Pretzel, P.

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Pringsheim, M.

Sequential Seizures in Neonates: Videos as a Diagnostic Tool for Diagnosis of KCNQ2-Related Self-Limiting Familial Neonatal Epilepsy?

Puthenparampil, M.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Quante, M.

Homozygous Frameshift Mutation in the ATP1A2 Gene Leading to Severe Pseudo-TORCH Syndrome

Radhakrishnan, K.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Raffler, S.

Primary Headache Types and Quality of Life in Children with Epilepsy

Rafii, P.

Expanding Clinical and Genetic Spectrum of IL-6 Signal Transduction Disorders Reveal Variable Immunodeficiency and Neurodevelopmental Disorders with Dysregulated Vesicular Trafficking

Rahman, F.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Rajbhandari, R.

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Ramanathan, S.

Treatment Response in Children with Relapsing MOGAD

Rauchenzauner, M.

Primary Headache Types and Quality of Life in Children with Epilepsy

Pulsatile Dexamethasone Therapy Reduces Epileptic Burden and Improves Sleep Physiology in Children with Genetic Drug-Resistant Epilepsy

Sleep Macro- and Microstructure Is Altered in Children with Epilepsy

Reihle, C.

Type 1 Interferon Activation in Mitochondrial Disease: A Possible “New” Pathomechanism

Reindl, M.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Treatment Response in Children with Relapsing MOGAD

Reiter-Fink, E.

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Reupero, S.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Reyes, E. de los

Cerliponase Alfa for the Treatment of CLN2 Disease in a Patient Cohort Including Children under 3 Years

Richardson, A.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Roloff, S.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Rooney, T.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Rosenbaum, T.

Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1)

Rosewich, H.

Region-Specific Lipidomic Profiling in Brain and Spinal Cord Tissue of X-Linked Adrenoleukodystrophy Mice

Regional White Matter Hypertrophy Follows Cerebral ABCD1 Gene Expression Pattern in Asymptomatic X-Linked Adrenoleukodystrophy

Rosso, M.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Rostasy, K.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Treatment Response in Children with Relapsing MOGAD

Influence of Fingolimod Treatment on Disease Outcome and MRI Brain Volumes in Children with CLN 3

Acute Unilateral Mydriasis in a Previously Healthy 10-Year-Old Girl: Differential Diagnoses

Rostásy, K.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Roubertie, A.

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Rüsch, C. T.

Impact of Preventable Respiratory Tract Infections on Spinal Muscular Atrophy with Focus on Respiratory Syncytial Virus Infections: A Retrospective Single-Center Study

Russell, A.

Eladocagene Exuparvovec Gene Therapy Increases Bayley-III Cognitive and Language Raw Scores in Patients with Aromatic ʟ-Amino Acid Decarboxylase Deficiency

Ryvlin, P.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Saffari, A.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Sahm, F.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Sahoo, P.

Regional White Matter Hypertrophy Follows Cerebral ABCD1 Gene Expression Pattern in Asymptomatic X-Linked Adrenoleukodystrophy

Sajeev, G.

Long-Term Comparative Efficacy and Safety of Risdiplam versus Nusinersen in Children with Type 1 Spinal Muscular Atrophy (SMA)

Salandin, M.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Salayev, K.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Santer, R.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Sartori, S.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Saunders, C.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Sauteur, P. M. Meyer

Impact of Preventable Respiratory Tract Infections on Spinal Muscular Atrophy with Focus on Respiratory Syncytial Virus Infections: A Retrospective Single-Center Study

Saxena, S.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Schanda, K.

Treatment Response in Children with Relapsing MOGAD

Schara, U.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Scharf, F.

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Scheller, J.

Expanding Clinical and Genetic Spectrum of IL-6 Signal Transduction Disorders Reveal Variable Immunodeficiency and Neurodevelopmental Disorders with Dysregulated Vesicular Trafficking

Schiller, K.

Primary Headache Types and Quality of Life in Children with Epilepsy

Pulsatile Dexamethasone Therapy Reduces Epileptic Burden and Improves Sleep Physiology in Children with Genetic Drug-Resistant Epilepsy

Sleep Macro- and Microstructure Is Altered in Children with Epilepsy

Schiller, V.

Primary Headache Types and Quality of Life in Children with Epilepsy

Schimmel, M.

Primary Headache Types and Quality of Life in Children with Epilepsy

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Treatment Response in Children with Relapsing MOGAD

Schlotawa, L.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Roifman Syndrome Is a Rare but Important Differential Diagnosis in Patients Suspected to Have CDG Syndrome

Schmid, R.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Schmidt, M.

Expanding Clinical and Genetic Spectrum of IL-6 Signal Transduction Disorders Reveal Variable Immunodeficiency and Neurodevelopmental Disorders with Dysregulated Vesicular Trafficking

Schmidt, U. Schara

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Schmitt, D.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Schnaufer, L.

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Long-Term Negative Impact of Pediatric Stroke on Language

Schödl, C.

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Schoenberg, N.

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Scholz, M.

Efficient Molecular Genetic Diagnostics in Tuberous Sclerosis Complex (TSC) by Deep Next-Generation Sequencing (NGS) of the Entire Genomic TSC Gene Regions Discovers Challenging Variants

Scholz, V.

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Schönberg, N.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Schönberger, J.

Neocortical and Mesial Temporal Sleep Spindles Are Reduced in Children with Focal Epilepsy and Developmental Delay

Schoonjans, A.-S.

Effect of Fenfluramine on Generalized Tonic–Clonic Seizures in Rare Epilepsy Syndromes: A Review of Published Studies

Schramm, C.

Benign Tonic Downgaze of Infancy (Pseudo-Sunset Phenomenon)

Schramm, S.

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Schreiber, G.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Schröder, S.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Schubert-Bast, S.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Schülke, M.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Schulz, A.

Cerliponase Alfa for the Treatment of CLN2 Disease in a Patient Cohort Including Children under 3 Years

Schulze-Bonhage, A.

Neocortical and Mesial Temporal Sleep Spindles Are Reduced in Children with Focal Epilepsy and Developmental Delay

Schumann, A.

Type 1 Interferon Activation in Mitochondrial Disease: A Possible “New” Pathomechanism

Schüssler, S.

Elevated TNF-α Levels and CD4 Cell Counts in the Blood of Children with Tuberous Sclerosis Complex (TSC)-Related Refractory Epilepsy

Schwartz, O.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Schwarz, D.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Schwendener, A.

Distinction of Cognitive Phenotypes in Pediatric-Onset Multiple Sclerosis

Scott, D. A.

Long-Term Comparative Efficacy and Safety of Risdiplam versus Nusinersen in Children with Type 1 Spinal Muscular Atrophy (SMA)

Seibert, J.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Seidl, R.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Sendelbach, K.

Investigation of Optical Genome Mapping Diagnostic Capabilities as a Potential Routine Clinical Test

Servais, L.

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

Shah, V.

Treatment Response in Children with Relapsing MOGAD

Sierra, J. R.

Eladocagene Exuparvovec Gene Therapy Increases Bayley-III Cognitive and Language Raw Scores in Patients with Aromatic ʟ-Amino Acid Decarboxylase Deficiency

Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment

Slasor, P.

Cerliponase Alfa for the Treatment of CLN2 Disease in a Patient Cohort Including Children under 3 Years

So, J.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

So, K.

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Sollmann, N.

Muscular Hyperalgesia in Young Adults with Migraine Is Associated with Headache Attacks

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

Sommer, S.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Specchio, N.

Providing Quality Care for People with CDKL5 Deficiency Disorder (CDD): An Expert Panel Opinion on the European Patient Journey

Cerliponase Alfa for the Treatment of CLN2 Disease in a Patient Cohort Including Children under 3 Years

Spiekerkoetter, U.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Stadelmann, C.

Region-Specific Lipidomic Profiling in Brain and Spinal Cord Tissue of X-Linked Adrenoleukodystrophy Mice

Statland, J.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Staudt, M.

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Long-Term Negative Impact of Pediatric Stroke on Language

Steigleder, L.

MRI Imaging in 10 Children with Enterovirus-Associated Acute Flaccid Myelitis

Stenzel, W.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Stettner, G.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Stettner, G. M.

Impact of Preventable Respiratory Tract Infections on Spinal Muscular Atrophy with Focus on Respiratory Syncytial Virus Infections: A Retrospective Single-Center Study

Stöffler, L.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Strozzi, S.

The Swiss Pediatric Inflammatory Brain Disease Cohort Study: First Insights into Epidemiology

Sturz, M.

Impact of Preventable Respiratory Tract Infections on Spinal Muscular Atrophy with Focus on Respiratory Syncytial Virus Infections: A Retrospective Single-Center Study

Stüve, B.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Sudheeran, K.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Sultan, T.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Sun, P.

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Sutherland, C. S.

Long-Term Comparative Efficacy and Safety of Risdiplam versus Nusinersen in Children with Type 1 Spinal Muscular Atrophy (SMA)

Tacke, M.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

Tai, C.-H.

Eladocagene Exuparvovec Gene Therapy Increases Bayley-III Cognitive and Language Raw Scores in Patients with Aromatic ʟ-Amino Acid Decarboxylase Deficiency

Takeshima, Y.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Tam, A.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Thamm, A.

Short-Term Effects of Selective Dorsal Rhizotomy on Gait in Children with Bilateral Spastic Cerebral Palsy

Thiels, C.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Treatment Response in Children with Relapsing MOGAD

Thimm, E.

Neurological Outcome in Screened Individuals with LCHAD/MTP Deficiency

Thomale, U. W.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Thomas, J.

Pulsatile Dexamethasone Therapy Reduces Epileptic Burden and Improves Sleep Physiology in Children with Genetic Drug-Resistant Epilepsy

Thomas, K.

Liquid Biopsy: Short-Cut to Identify Mosaic Causes of Hemihypertrophic Overgrowth

Tietze, A.

Atypical NMDA Receptor Expression in a Diffuse Astrocytoma, MYB- or MYBL1-Altered as a Trigger for Autoimmune Encephalitis

Weathering the Storm: Early Intervention for GAD65 Antibody-Mediated Encephalitis with Drug-Resistant Autoimmune Epilepsy

A Deceptive Bump: The Case of a Complicated Pott's Puffy Tumor in a Symptom-Free Child

Toepffer, E. M.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Torbati, P. Najarzadeh

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Trifillis, P.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Trollmann, R.

Elevated TNF-α Levels and CD4 Cell Counts in the Blood of Children with Tuberous Sclerosis Complex (TSC)-Related Refractory Epilepsy

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Trück, J.

Impact of Preventable Respiratory Tract Infections on Spinal Muscular Atrophy with Focus on Respiratory Syncytial Virus Infections: A Retrospective Single-Center Study

Ullah, W.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Ullmann, F.

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Unterholzner, G.

Primary Headache Types and Quality of Life in Children with Epilepsy

Pulsatile Dexamethasone Therapy Reduces Epileptic Burden and Improves Sleep Physiology in Children with Genetic Drug-Resistant Epilepsy

Sleep Macro- and Microstructure Is Altered in Children with Epilepsy

Vandenborne, K.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Givinostat in Duchenne Muscular Dystrophy: Effect on Disease Milestones

van Elten, A.

Influence of Fingolimod Treatment on Disease Outcome and MRI Brain Volumes in Children with CLN 3

Viskochil, D.

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Volk, A.

Description of the Hamburg Alexander Leukodystrophy Cohort

von Both, U.

Childhood Stroke as a Challenging Complication in Severe Systemic Mycosis

von der Hagen, M.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Wagner, J.

Feasibility of an Instrumented Gait Testing Protocol for Children after Mild Traumatic Brain Injury

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

2B or Not 2B: Results of the Study 2B in Motion Examining Balance after Mild Traumatic Brain Injury in Children and Adolescents

Wagner, K.

Neocortical and Mesial Temporal Sleep Spindles Are Reduced in Children with Focal Epilepsy and Developmental Delay

Wagner, M.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Walter, G.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Waltz, S.

Clinical and Imaging Features of Children with Autoimmune Encephalitis and GFAP Antibodies

Wandinger, K.-P.

Frequency of Autoantibodies against the Gray and White Matter in Children with Suspected Encephalitis in the MERIN Study

Wang, A.

Eladocagene Exuparvovec Gene Therapy Increases Bayley-III Cognitive and Language Raw Scores in Patients with Aromatic ʟ-Amino Acid Decarboxylase Deficiency

Waxman, E. A.

Tazarotene and Bexarotene Show Efficacy as In Vitro Therapeutic Agents in Multiple Sulfatase Deficiency

Weber, K.

Sleep Macro- and Microstructure Is Altered in Children with Epilepsy

Weber-Ferro, W.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Weckhuysen, S.

Sequential Seizures in Neonates: Videos as a Diagnostic Tool for Diagnosis of KCNQ2-Related Self-Limiting Familial Neonatal Epilepsy?

Wegener-Panzer, A.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Weigel, C.

Metabolic Neuropathies in Children and Adolescents with LCHAD/MTP Deficiency: Insights from In Vivo Magnetic Resonance Neurography

Weiss, K.

Delineation of Laminopathies as Progeroid and Neurodevelopmental Disorders Due to Deficient Nuclear Membrane Trafficking

Weiß, C.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Wels, A.

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Wendel, E. M.

A20 (TNFAIP3) Distinguishes Relapse from Remission in Pediatric Patients with Monophasic MOGAD: Preliminary Results

Outcome and Brain Volume Changes over Time in Children with Autoimmune Encephalitis and MOG Antibodies

Wendel, E.-M.

IgM Synthesis and MRZ Reaction Are Not Associated with Factors Predicting a Poor Prognosis in Pediatric MS

Acute Disseminated Encephalomyelitis without MOG Antibodies: Clinical Course and Final Diagnosis

Long-Term Follow-up of Children with Pre- and (Post) Pubertal MS

Treatment Response in Children with Relapsing MOGAD

Treatment Response in Children with Relapsing MOGAD

Werner, C.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Wiechers, C.

Homozygous Frameshift Mutation in the ATP1A2 Gene Leading to Severe Pseudo-TORCH Syndrome

Wiese, M. L.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Wiethoff-Ubrig, L.

Acute Unilateral Mydriasis in a Previously Healthy 10-Year-Old Girl: Differential Diagnoses

Wilcocks, R.

Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to MR Measures of Muscle Fat Fraction

Wilichowski, E.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Wilke, M.

Structural Changes in the Corticospinal Tract of the Contralesional Hemisphere following Perinatal Lesions

Long-Term Negative Impact of Pediatric Stroke on Language

Williams, P.

Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials

Winklmeier, S.

Study of the Lymphocyte Profile in Highly Active Pediatric Multiple Sclerosis Patients before and after Therapy with Fingolimod

Witzel, M.

Liquid Biopsy: Short-Cut to Identify Mosaic Causes of Hemihypertrophic Overgrowth

Wohlfrom, T.

Detection of 5q-Spinal Muscular Atrophy by Short- and Long-Read Sequencing

Würzl, J.

Sleep Macro- and Microstructure Is Altered in Children with Epilepsy

Wysocki, M.

A Phase 1 Study to Assess the Effect of Food on the Pharmacokinetics (PK) and Gastrointestinal (GI) Tolerability of Selumetinib in Adolescents with Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PN)

Yu, J.

Biallelic Variants in OAS2 with Neurodevelopmental Disorders, Skeletal Dysplasia, and Immunodeficiency

Zaddach, M.

PRiSMA—Pediatric Registry for Stroke as a Multidisciplinary Approach to Healthcare Research

Zaidenstadt, E.

Benefits of Repetitive Neuromuscular Magnetic Stimulation in Pediatric Posttraumatic Headache

Zaidman, C.

Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy: Interim Findings from the Phase 3 SMART Study

Zaki, M. S.

Expanding SNX14-Associated Movement Disorders in a Genotype–Phenotype Spectrum

Zamani, M.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders

Zambon, A.

Long-Term Outcome of Gene Therapy for MLD and Prospective Newborn Screening: The Tübingen Experience

Ziegler, A.

Real-World Data on the Efficacy of Gene Replacement Therapy for Spinal Muscular Atrophy (SMA)

Zifarelli, G.

Biallelic Variants in OAS2 with Neurodevelopmental Disorders, Skeletal Dysplasia, and Immunodeficiency

Zirn, B.

Expanding the Clinical and Genetic Spectrum of RAB3GAP1-Related Disorders with Novel Movement Disorders