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Semin Neurol 2025; 45(02): 287-297
DOI: 10.1055/a-2580-1187
Review Article

The Epilepsy Drug Pipeline: Update on Near-to-Market Therapies

Christopher Elder 
1   Department of Neurology, NYU Grossman School of Medicine and NYU Langone Health, New York, New York
,
Jacob Pellinen 
2   Department of Neurology, University of Colorado School of Medicine, Aurora, Colorado
,
Samuel W. Terman 
3   Department of Neurology, University of Michigan, Ann Arbor, Michigan
› Author Affiliations
Funding Pharmaceutical companies were allowed review and approval of the corresponding sections of this manuscript. C.E. serves as a paid speaker for SK Life Science. S.W.T. was supported by the National Institutes of Health K23 AG081463. He has been a paid consultant for Jazz Pharmaceuticals and is currently a paid consultant for Xenon Pharmaceuticals. J.P. has received research support from the Department of Neurology at the University of Colorado School of Medicine, the Colorado Clinical and Translational Sciences Institute by way of NIH/NCATS Colorado CTSA Grant Number UL1 TR002535, NIH/NINDS, the American Epilepsy Society, and SK Life Science. He has done consulting work and/or attended Scientific Advisory Boards for SK Life Science and Jazz Pharmaceuticals. He is the Chair of the Professional Advisory Board for the Epilepsy Foundation of Colorado and Wyoming and serves on the Professional Advisory Board for the Epilepsy Foundation of America. He is the Epilepsy Section Editor for Current Neurology and Neuroscience Reports.
› Further Information
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Abstract

Since the first antiseizure medication (ASM) was introduced in 1857, more than 30 medications have been approved by the United States Food and Drug Administration (FDA) for the treatment of epilepsy. However, limitations in efficacy and tolerability have led to one-third of patients suffering from uncontrolled seizures. Recent advances in genetics, disease modeling, high-throughput target-based and phenotype-based screening, study design, and identification of novel mechanisms of action or routes of delivery have resulted in more than 200 therapeutics currently under development in the epilepsy pipeline. This study discusses near-to-market drugs in advanced clinical development, with select drugs in earlier stages. Background regarding mechanisms, animal studies, pharmacokinetics, pharmacodynamics, efficacy, tolerability, and safety data are provided for each drug when available.

Keywords

epilepsy - antiseizure medications - clinical trials - pipeline

Authors' Contributions

All authors contributed equally to the writing and editing of this manuscript.




Publication History

Article published online:
08 May 2025

© 2025. Thieme. All rights reserved.

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  • References

  • 1 Klein P, Krauss GL, Steinhoff BJ, Devinsky O, Sperling MR. Failure to use new breakthrough treatments for epilepsy. Epilepsia 2023; 64 (06) 1458-1465
    PubMedSearch in Google Scholar
  • 2 Chen Z, Brodie MJ, Liew D, Kwan P. Treatment outcomes in patients with newly diagnosed epilepsy treated with established and new antiepileptic drugs: a 30-year longitudinal cohort study. JAMA Neurol 2018; 75 (03) 279-286
    CrossrefPubMedSearch in Google Scholar
  • 3 Jakus R, Bagdy G. The Role of 5–HT2C Receptor in Epilepsy. In: 2011: 429-444
    Search in Google Scholar
  • 4 Griffin A, Hamling KR, Knupp K, Hong S, Lee LP, Baraban SC. Clemizole and modulators of serotonin signalling suppress seizures in Dravet syndrome. Brain 2017; 140 (03) 669-683
    PubMedSearch in Google Scholar
  • 5 Bialer M, Johannessen SI, Koepp MJ. et al. Progress report on new medications for seizures and epilepsy: A summary of the 17th Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVII). II. Drugs in more advanced clinical development. Epilepsia 2024; 65 (10) 2858-2882
    PubMedSearch in Google Scholar
  • 6 Parasrampuria D, Mills I, O'Connell G, Chaudhary A, Ruckle J, Orevillo C. . A randomized, double- blind, placebo-controlled, multiple ascending dose (MAD) pharmacokinetics (PK), pharmacodynamics (PD), and tolerability study of LP352 in healthy subjects [abstract 10–022]. Poster Presented at: American Academy of Neurology (AAN) Annual Meeting; April 2–7th, 2022; Seattle Washington; April 24–26th, 2022 . Accessed January 10, 2025 at: https://www.aan.com/MSA/Public/Events/AbstractDetails/50543
    PubMed
  • 7 Orevillo C, Bialek S, Dlugos D, French J, Kaye R. The PACIFIC study: a phase 1b/2a study to investigate the safety, tolerability, pharmacology, and exploratory efficacy of LP352 in subjects with developmental and epileptic encephalopathies [abstract 758]. Poster Presented at: 35th International Epilepsy Congress (IEC), September 2–6th, 2023 , Dublin, Ireland. Accessed January 10, 2025 at: https://www.ilae.org/files/dmfile/iec-2023-abstract-book-for-website-11823.pdf
    PubMedSearch in Google Scholar
  • 8 Elks J. Clemizole. In: Ganellin CR, Elks J. eds. The Dictionary of Drugs: Chemical Data. Vol 1. 1st ed. Springer US; 1990: 288-289
    Search in Google Scholar
  • 9 Griffin AL, Jaishankar P, Grandjean JM, Olson SH, Renslo AR, Baraban SC. Zebrafish studies identify serotonin receptors mediating antiepileptic activity in Dravet syndrome. Brain Commun 2019; 1 (01) fcz008
    PubMedSearch in Google Scholar
  • 10 Baraban SC, Dinday MT, Hortopan GA. Drug screening in Scn1a zebrafish mutant identifies clemizole as a potential Dravet syndrome treatment. Nat Commun 2013; 4: 2410
    PubMedSearch in Google Scholar
  • 11 Rao L, Masuoka L, Shatzoff M, Lee HJ, Baraban S. EPX-100 as an Adjunctive Therapy in Dravet Syndrome: Phase 1 and Phase 2 Randomized, Double-blind, Placebo-controlled Trials. Poster Presented at: American Epilepsy Society (AES) Annual Meeting; December 4th, 2022 ; Nashville, Tennessee
    PubMedSearch in Google Scholar
  • 12 Gogas KR. Glutamate-based therapeutic approaches: NR2B receptor antagonists. Curr Opin Pharmacol 2006; 6 (01) 68-74
    PubMedSearch in Google Scholar
  • 13 Mony L, Kew JNC, Gunthorpe MJ, Paoletti P. Allosteric modulators of NR2B-containing NMDA receptors: molecular mechanisms and therapeutic potential. Br J Pharmacol 2009; 157 (08) 1301-1317
    PubMedSearch in Google Scholar
  • 14 Hanada T. Ionotropic glutamate receptors in epilepsy: a review focusing on AMPA and NMDA receptors. Biomolecules 2020; 10 (03) 464
    PubMedSearch in Google Scholar
  • 15 Mullier B, Wolff C, Sands ZA. et al. GRIN2B gain of function mutations are sensitive to radiprodil, a negative allosteric modulator of GluN2B-containing NMDA receptors. Neuropharmacology 2017; 123: 322-331
    CrossrefPubMedSearch in Google Scholar
  • 16 Alkhachroum A, Der-Nigoghossian CA, Mathews E. et al. Ketamine to treat super-refractory status epilepticus. Neurology 2020; 95 (16) e2286-e2294
    CrossrefPubMedSearch in Google Scholar
  • 17 Amador A, Bostick CD, Olson H. et al. Modelling and treating GRIN2A developmental and epileptic encephalopathy in mice. Brain 2020; 143 (07) 2039-2057
    PubMedSearch in Google Scholar
  • 18 Bertocchi I, Cifarelli L, Oberto A. et al. Radiprodil, a selective GluN2B negative allosteric modulator, rescues audiogenic seizures in mice carrying the GluN2A(N615S) mutation. Br J Pharmacol 2024; 181 (12) 1886-1894
    PubMedSearch in Google Scholar
  • 19 Auvin S, Dozières-Puyravel B, Avbersek A. et al. Radiprodil, a NR2B negative allosteric modulator, from bench to bedside in infantile spasm syndrome. Ann Clin Transl Neurol 2020; 7 (03) 343-352
    PubMedSearch in Google Scholar
  • 20 GRIN Therapeutics Announces Positive Topline Data from Honeycomb Trial of Radiprodil in GRIN-Related Neurodevelopmental Disorder. . News release. September 9, 2024 . Accessed December 11, 2024 at: https://www.prnewswire.com/news-releases/grin-therapeutics-announces-positive-topline-data-from-honeycomb-trial-of-radiprodil-in-grin-related-neurodevelopmental-disorder-302240847.html
    PubMed
  • 21 Zhang D, Lape R, Shaikh SA. et al. Modulatory mechanisms of TARP γ8-selective AMPA receptor therapeutics. Nat Commun 2023; 14 (01) 1659
    PubMedSearch in Google Scholar
  • 22 Maher MP, Wu N, Ravula S. et al. Discovery and characterization of AMPA receptor modulators selective for TARP-γ8. J Pharmacol Exp Ther 2016; 357 (02) 394-414
    PubMedSearch in Google Scholar
  • 23 Coombs ID, Cull-Candy SG. Transmembrane AMPA receptor regulatory proteins and AMPA receptor function in the cerebellum. Neuroscience 2009; 162 (03) 656-665
    PubMedSearch in Google Scholar
  • 24 Maher MP, Matta JA, Gu S, Seierstad M, Bredt DS. Getting a handle on neuropharmacology by targeting receptor-associated proteins. Neuron 2017; 96 (05) 989-1001
    PubMedSearch in Google Scholar
  • 25 Bialer M, Johannessen SI, Koepp MJ. et al. Progress report on new medications for seizures and epilepsy: a summary of the 17th Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVII). I. Drugs in preclinical and early clinical development. Epilepsia 2024; 65 (10) 2831-2857
    PubMedSearch in Google Scholar
  • 26 Therapeutics R. Rapport Therapeutics Announces New Phase 1 Data, Further Supporting RAP-219's Transformative Potential for CNS Disorders. https://investors.rapportrx.com/news-releases/news-release-details/rapport-therapeutics-announces-new-phase-1-data-further . January 9, 2025 . Accessed September 30, 2025 at: https://investors.rapportrx.com/news-releases/news-release-details/rapport-therapeutics-announces-new-phase-1-data-further
    PubMedSearch in Google Scholar
  • 27 Scharf SH, Jaeschke G, Wettstein JG, Lindemann L. Metabotropic glutamate receptor 5 as drug target for Fragile X syndrome. Curr Opin Pharmacol 2015; 20: 124-134
    PubMedSearch in Google Scholar
  • 28 Li Y, Corradetti MN, Inoki K, Guan KL. TSC2: filling the GAP in the mTOR signaling pathway. Trends Biochem Sci 2004; 29 (01) 32-38
    CrossrefPubMedSearch in Google Scholar
  • 29 Noema Pharma. Our Pipeline: Basimglurant (NOE-101). Accessed January 10, 2025 at: https://noemapharma.com/pipeline/#NOE-101
    PubMed
  • 30 Lindemann L, Porter RH, Scharf SH. et al. Pharmacology of basimglurant (RO4917523, RG7090), a unique metabotropic glutamate receptor 5 negative allosteric modulator in clinical development for depression. J Pharmacol Exp Ther 2015; 353 (01) 213-233
    PubMedSearch in Google Scholar
  • 31 Quiroz JA, Tamburri P, Deptula D. et al. Efficacy and safety of basimglurant as adjunctive therapy for major depression: a randomized clinical trial. JAMA Psychiatry 2016; 73 (07) 675-684
    PubMedSearch in Google Scholar
  • 32 Youssef EA, Berry-Kravis E, Czech C. et al; FragXis Study Group. Effect of the mGluR5-NAM basimglurant on behavior in adolescents and adults with fragile X syndrome in a randomized, double-blind, placebo-controlled trial: FragXis phase 2 results. Neuropsychopharmacology 2018; 43 (03) 503-512
    PubMedSearch in Google Scholar
  • 33 Allen MJ, Sabir S, Sharma S. GABA receptor. Trends Pharmacol Sci 2023; 2 (C): 62-64
    PubMedSearch in Google Scholar
  • 34 Sarmiere PD, Colburn R, Mukherjee J. Evaluation of OV329, a next-generation GABA-AT inhibitor in a series of Pharmaco- resistant seizure models through the NINDS epilepsy therapy screening program. Annual Meeting of the American Epilepsy Society (abstract), Nashville, Tennessee. Published online 2022
    PubMed
  • 35 Sarmiere PD, Roucard C, Evrard A, Fishback J. OV329, a GABA aminotransferase (GABA-AT) Inhibitor, suppresses hippocampal paroxysmal discharges in a human translational mesial-temporal lobe epilepsy (MTLE) mouse model. Annual Meeting of the American Epilepsy Society, Chicago, (abstract). Published online 2021
    PubMed
  • 36 Feja M, Meller S, Deking LS. et al. OV329, a novel highly potent γ-aminobutyric acid aminotransferase inactivator, induces pronounced anticonvulsant effects in the pentylenetetrazole seizure threshold test and in amygdala-kindled rats. Epilepsia 2021; 62 (12) 3091-3104
    PubMedSearch in Google Scholar
  • 37 Terman SW, Kirkpatrick L, Akiyama LF. et al. Current state of the epilepsy drug and device pipeline. Epilepsia 2024; 65 (04) 833-845
    PubMedSearch in Google Scholar
  • 38 Hoy SM. Ganaxolone: a review in epileptic seizures associated with cyclin-dependent kinase-like 5 deficiency disorder. Paediatr Drugs 2025; 27 (01) 111-118
    PubMedSearch in Google Scholar
  • 39 Reddy DS. Neurosteroids as novel anticonvulsants for refractory status epilepticus and medical countermeasures for nerve agents: a 15-year journey to bring ganaxolone from bench to clinic. J Pharmacol Exp Ther 2024; 388 (02) 273-300
    PubMedSearch in Google Scholar
  • 40 Gasior M, Husain A, Barra ME. et al. Intravenous ganaxolone: pharmacokinetics, pharmacodynamics, safety, and tolerability in healthy adults. Clin Pharmacol Drug Dev 2024; 13 (03) 248-258
    PubMedSearch in Google Scholar
  • 41 Vaitkevicius H, Ramsay RE, Swisher CB, Husain AM, Aimetti A, Gasior M. Intravenous ganaxolone for the treatment of refractory status epilepticus: results from an open-label, dose-finding, phase 2 trial. Epilepsia 2022; 63 (09) 2381-2391
    CrossrefPubMedSearch in Google Scholar
  • 42 Lamb YN. Ganaxolone: first approval. Drugs 2022; 82 (08) 933-940
    PubMedSearch in Google Scholar
  • 43 Foreman B. Efficacy and Safety of Intravenous Ganaxolone for Treatment of Refractory Status Epilepticus: Results from the Phase 3, Double-Blind, Randomized, Placebo-Controlled RAISE Trial. Neurocritical Care Society Annual Meeting October 17th 2024 San Diego, California
    PubMedSearch in Google Scholar
  • 44 Löscher W. Novel antiseizure medications in the development pipeline: promising candidates and recent failures. Clin Epileptol 2025; 38: 42-53
    PubMedSearch in Google Scholar
  • 45 Anderson L. PRAX-562 is a well-tolerated, novel persistent sodium channel blocker with broad anticonvulsant activity in multiple DEE mouse models (S34.007). Neurology. 2023;100(17 suppl):100(17_Supplement)2
    PubMed
  • 46 Pfister B, Spar B, Dalby K. et al. EMBOLD: A Clinical Trial of PRAX-562 in Subjects with Developmental and Epileptic Encephalopathies Followed by an Open-Label Extension. Presented at: American Epilepsy Society (AES) Annual Meeting; December 2023; Orlando, Florida. Accessed January 10, 2025 at: https://praxismedicines.com/wp-content/uploads/2023/12/Pfister_AES2023_EMBOLD_Poster_Final.pdf
    PubMed
  • 47 Praxis Precision Medicines announces positive topline results from the EMBOLD study in SCN2A and 8A developmental epilepsies, highlighting the disease-modifying potential of relutrigine. Accessed January 10, 2025 at: https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-announces-positive-topline-results-0
    PubMed
  • 48 Pong AW, Ross J, Tyrlikova I. et al. Epilepsy: expert opinion on emerging drugs in phase 2/3 clinical trials. Expert Opin Emerg Drugs 2022; 27 (01) 75-90
    PubMedSearch in Google Scholar
  • 49 Novak GP, Keating PE, Wamil AW, Nadler JV, Donevan SD. Carisbamate (RWJ-333369). Neurotherapeutics 2007; 4 (01) 106-109
    PubMedSearch in Google Scholar
  • 50 Faught E, Holmes GL, Rosenfeld WE. et al. Randomized, controlled, dose-ranging trial of carisbamate for partial-onset seizures. Neurology 2008; 71 (20) 1586-1593
    PubMedSearch in Google Scholar
  • 51 Halford JJ, Sperling MR, Arkilo D. et al. A phase 1b/2a study of soticlestat as adjunctive therapy in participants with developmental and/or epileptic encephalopathies. Epilepsy Res 2021; 174: 106646
    PubMedSearch in Google Scholar
  • 52 Sperling MR, Greenspan A, Cramer JA. et al. Carisbamate as adjunctive treatment of partial onset seizures in adults in two randomized, placebo-controlled trials. Epilepsia 2010; 51 (03) 333-343
    PubMedSearch in Google Scholar
  • 53 Lu C, Zheng J, Cao Y, Bresnahan R, Martin-McGill KJ. Carisbamate add-on therapy for drug-resistant focal epilepsy. Cochrane Database Syst Rev 2021; 12 (12) CD012121
    PubMedSearch in Google Scholar
  • 54 Faulkner IE, Pajak RZ, Harte MK, Glazier JD, Hager R. Voltage-gated potassium channels as a potential therapeutic target for the treatment of neurological and psychiatric disorders. Front Cell Neurosci 2024; 18: 1449151
    PubMedSearch in Google Scholar
  • 55 Khan R, Chaturvedi P, Sahu P. et al. Role of potassium ion channels in epilepsy: focus on current therapeutic strategies. CNS Neurol Disord Drug Targets 2024; 23 (01) 67-87
    PubMedSearch in Google Scholar
  • 56 Costi S, Morris LS, Kirkwood KA. et al. Impact of the KCNQ2/3 channel opener ezogabine on reward circuit activity and clinical symptoms in depression: results from a randomized controlled trial. Am J Psychiatry 2021; 178 (05) 437-446
    PubMedSearch in Google Scholar
  • 57 French JA, Porter RJ, Perucca E. et al; X-TOLE Study Group. Efficacy and safety of XEN1101, a novel potassium channel opener, in adults with focal epilepsy: a phase 2b randomized clinical trial. JAMA Neurol 2023; 80 (11) 1145-1154
    PubMedSearch in Google Scholar
  • 58 French J, Porter R, Perucca E. et al. Interim Long-Term Safety and Efficacy of XEN1101, a Potent, Selective Potassium Channel Opener: Update from an Ongoing, Open-Label Extension of a Phase 2b Study (X-TOLE) in Adults with Focal Epilepsy. Annual Meeting of the American Epilepsy Society (AES) [abstract] Orlando, Florida December 2, 2023
    PubMedSearch in Google Scholar
  • 59 Greene DL, Hoshi N. Modulation of Kv7 channels and excitability in the brain. Cell Mol Life Sci 2017; 74 (03) 495-508
    PubMedSearch in Google Scholar
  • 60 Picchione K, Resnick L, Bozik M, Dworetzky S. Characterization of BHV-7000: A Novel Kv7.2/7.3 Activator for the Treatment of Seizures. Presented at American Epilepsy Society Annual Meeting December 3rd, 2023 Orlando, Florida
    PubMedSearch in Google Scholar
  • 61 Lerner J, Awsare B, Sevinsky E. et al. Novel, Selective Kv7.2/7.3 Potassium Channel Activator, BHV-7000, Demonstrates Dose-Dependent Pharmacodynamic Effects on EEG Parameters in Healthy Adults. Presented at American Epilepsy Society Annual Meeting Orlando, Florida December 3rd, 2023
    PubMedSearch in Google Scholar
  • 62 Klein P, Kaminski RM, Koepp M, Löscher W. New epilepsy therapies in development. Nat Rev Drug Discov 2024; 23 (09) 682-708
    PubMedSearch in Google Scholar
  • 63 Nishi T, Kondo S, Miyamoto M. et al. Soticlestat, a novel cholesterol 24-hydroxylase inhibitor shows a therapeutic potential for neural hyperexcitation in mice. Sci Rep 2020; 10 (01) 17081
    PubMedSearch in Google Scholar
  • 64 Hahn CD, Jiang Y, Villanueva V. et al. A phase 2, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of soticlestat as adjunctive therapy in pediatric patients with Dravet syndrome or Lennox-Gastaut syndrome (ELEKTRA). Epilepsia 2022; 63 (10) 2671-2683
    PubMedSearch in Google Scholar
  • 65 Yao Y. Long-term treatment effects of soticlestat in patients with Dravet syndrome or Lennox–Gastaut syndrome: interim data from the ENDYMION 1 trial (S44.004). Neurology 2023; 100 (17) 3045
    PubMedSearch in Google Scholar
  • 66 Gnatek Y, Zimmerman G, Goll Y, Najami N, Soreq H, Friedman A. Acetylcholinesterase loosens the brain's cholinergic anti-inflammatory response and promotes epileptogenesis. Front Mol Neurosci 2012; 5: 66
    PubMedSearch in Google Scholar
  • 67 Türkdönmez Ak E, Okuyucu B, Arslan G, Ağar E, Ayyildiz M. The role of acetylcholinesterase enzyme inhibitor rivastigmine on spike-wave discharges, learning-memory, anxiety, and TRPV1 channel expression in genetic absence epileptic WAG/Rij rats. Neurochem Res 2025; 50 (01) 67
    PubMedSearch in Google Scholar
  • 68 Casillas-Espinosa PM, Garcia-Olivares J, Li R. et al. Huperzine A suppresses absence seizures in the genetic absence epilepsy rat from Strasbourg (GAERS) model of genetic generalized epilepsy with absence seizures. Epilepsia Open 2024; 9 (05) 1826-1836
    PubMedSearch in Google Scholar
  • 69 Portelli J, Park L, Lujan B, Busse G, Rubin J, Nasser A. An open-label pilot trial assessing the safety and efficacy of SPN-817 (huperzine A extended-release) in adults with treatment-resistant focal impaired awareness seizures (P12-8.003). Neurology 2023; 100 (17 suppl): P12-8.003
    PubMedSearch in Google Scholar
  • 70 Supernus Announces Promising Interim Data from Ongoing Open-Label Phase 2a Study of SPN-817 in Epilepsy. May 24, 2024 . Supernus Announces Promising Interim Data from Ongoing Open-Label Phase 2a Study of SPN-817 in Epilepsy. Accessed December 13, 2025 at: https://ir.supernus.com/news-releases/news-release-details/supernus-announces-promising-interim-data-ongoing-open-label
    PubMedSearch in Google Scholar
  • 71 O'Brien T, French J, Mehta N, Maskeri N, Asubonteng K, Upadhyaya H. The RENAISSANCE Study: Interim results of a phase 2 study of SPN-817 in adults with treatment-resistant epilepsy. Poster 2376 presented at the American Epilepsy Society Annual Meeting in Los Angeles December 6–10, 2024
    PubMedSearch in Google Scholar
  • 72 Supernus Announces Third Quarter 2024 Financial Results - Supernus Pharmaceuticals. Accessed February 25, 2025 at: https://ir.supernus.com/news-releases/news-release-details/supernus-announces-third-quarter-2024-financial-results
    PubMedSearch in Google Scholar
  • 73 DiNuzzo M, Mangia S, Maraviglia B, Giove F. Does abnormal glycogen structure contribute to increased susceptibility to seizures in epilepsy?. Metab Brain Dis 2015; 30 (01) 307-316
    PubMedSearch in Google Scholar
  • 74 Armstrong D, Wong V. VAL-1221: A Phase II Candidate for Lafora Disease. Annual Meeting of the American Epilepsy Society (abstract); December 3rd, 2023 Orlando, Florida
    PubMedSearch in Google Scholar
  • 75 Kishnani P, Lachmann R, Mozaffar T. et al. Safety and efficacy of VAL-1221, a novel fusion protein targeting cytoplasmic glycogen, in patients with late-onset Pompe disease. Mol Genet Metab 2019; 126 (02) S85-S86
    PubMedSearch in Google Scholar
  • 76 Colpaert M, Singh PK, Donohue KJ. et al. Neurological glycogen storage diseases and emerging therapeutics. Neurotherapeutics 2024; 21 (05) e00446
    PubMedSearch in Google Scholar
  • 77 Olson CA, Vuong HE, Yano JM, Liang QY, Nusbaum DJ, Hsiao EY. The gut microbiota mediates the anti-seizure effects of the ketogenic diet. Cell 2018; 173 (07) 1728-1741.e13
    PubMedSearch in Google Scholar
  • 78 Licamele L, Demers D, Elmen L. et al. Safety, Tolerability, and Strain Kinetics of BL-001, an Orally Delivered Live Biotherapeutic Product, in a Phase 1 Study of Healthy Volunteers. Abstract Presentation at the American Epilepsy Society Annual Meeting December 8th, 2024 Los Angeles, CA
    PubMedSearch in Google Scholar
  • 79 Nowinski B, Elmen L, Licamele L, Demers D, Baroldi P, Reyes C. Reverse Translational Validation of Anti-epileptic Effect of Prototype BL-001 in a Preclinical Seizure Model from a Phase 0 Trial in Healthy Volunteers. Abstract Presentation at the American Epilepsy Society Annual Meeting December 8th, 2024 Los Angeles, CA
    PubMedSearch in Google Scholar