Showing 4 of 4 recruiting trials for “Congenital myasthenic syndrome”
RecruitingNCT06630650 ↗
A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes
👨⚕️ Christopher Grunseich, M.D., National Institute of Neurological Disorders and Stroke (NINDS)📍 1 site📅 Started May 2025View details ↗
Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution
🏥 The Foundation for Orthopaedics and Regenerative Medicine📍 3 sites📅 Started Jan 2025View details ↗
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
RecruitingNCT06078553 ↗
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
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