Congenital Myasthenic Syndrome
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) [Phase 1]
A Phase 1b study to investigate the safety and tolerability of ARGX-119 in adults with DOK7-congenital myasthenic syndrome (CMS) is currentl…
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
A natural history study in participants with congenital myasthenic syndromes caused by mutations in DOK7, MUSK, AGRN, or LRP4 is currently r…
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CLINICAL_TRIALS research · 2025-11-10Clinical Trial Update: Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution [Phase 1]
Phase 1 clinical trial exploring mesenchymal stem cell exosome solution for treatment of congenital myasthenic syndrome; currently recruitin…