DOI : 10.1055/s-00000041

Neuropediatrics

Issue S 02 · Volume 50 · September 2019 DOI: 10.1055/s-009-44190


Abstracts of the 45th Annual Meeting of the Society for Neuropediatrics
München, 11.09 – 14.09.2019

Chair: Prof. Dr. Martin Staudt, Pediatric Neurology, Neurorehabilitation and Epileptology, Schön Klinik Vogtareuth
Abstracts (HTML) List of Authors
GNP-FV14
Schultz, Meredith; Swoboda, Kathryn J.; Farrar, Michelle; McMillan, Hugh; Parsons, Julie; Ernst, Uwe; Kernbauer, Elaine; Farrow, Marcia; Ogrinc, Francis G.; Kavanagh, Sarah; Feltner, Douglas E.; McGill, Bryan E.; Spector, Sidney A.; L’Italien, James; Sproule, Douglas; Strauss, Kevin: Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) in Pre-symptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update
GNP-FV15
Day, John W.; Chiriboga, Claudia A.; Crawford, Thomas O.; Darras, Basil T.; Finkel, Richard S.; Connolly, Anne M.; Iannaccone, Susan T.; Kuntz, Nancy L.; Peña, Loren; Schultz, Meredith; Shieh, Perry B.; Smith, Edward C.; Ernst, Uwe; Feltner, Douglas E.; Orgrinc, Francis G.; Shah, Ankita; Ouyang, Haojun; Macek, Thomas A.; Kernbauer, Elaine; L’Italien, James; Sproule, Douglas M.; Kaspar, Brian K.; Medel, Jerry R.: Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update
GNP-FV16
Muntoni, Francesco; Baranello, Giovanni; Bruno, Claudio; Corti, Stefania; Masson, Riccardo; Straub, Volker; Vita, Giuseppe; Ernst, Uwe; Kernbauer, Elaine; Williamson, Susan; Ouyang, Haojun; Feltner, Douglas E.; Meriggioli, Matthew; Lavrov, Arseniy; L’Italien, James; Sproule, Douglas M.; Mercuri, Eugenio: Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe
GNP-FV17
Müller-York, André; Iff, Joel; Gerrits, Charles; Sajeev, Gautam; Signorovitch, James; Tuttle, Edward; Birk, Erica; Hossain, Intekhab; Yao, Zhiwen; Ng, Courtney; Goemans, Nathalie: Eteplirsen Delays Time to Loss of Ambulation in Patients With Duchenne Muscular Dystrophy Compared With Patients Receiving Standard of Care
GNP-FV18
Regensburger, Adrian P.; Fonteyne, Lina; Jüngert, Jörg; Qurashi, Matthias; Neurath, Markus F.; Klymiuk, Nikolai; Kemter, Elisabeth; Rascher, Wolfgang; Trollmann, Regina; Wolf, Eckhard; Waldner, Maximilian J.; Knieling, Ferdinand: New Non-invasive Biomarkers in Duchenne Muscular Dystrophy: Translational Molecular Imaging with Multispectral Optoacoustic Tomography
GNP-FV19
Zielonka, Matthias; Garbade, Sven F.; Kölker, Stefan; Hoffmann, Georg F.; Ries, Markus: A Cross-sectional Quantitative Analysis of the Natural History of Alpha-mannosidosis
GNP-FV20
Polster, Tilman; Schaefer, Susanne; Panzer, Axel: Visual Snow - Two Adolescents with a New Syndrome Different from Migraine or Epilepsy
GNP-FV021a
Raile, Vera; Herz, Nina; Promnitz, Gabriel; Schneider, Joanna; Kaindl, Angela Maria: Clinical Outcome of Children with Corpus Callosum Agenesis