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Myasthenic syndrome, congenital, 24, presynaptic

MONDO:0032597

Also known as: CMS24, MYASTHENIC SYNDROME, CONGENITAL, 24, PRESYNAPTIC

1 clinical trial for this condition and its sub-types.

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Broader categories

Disease (618) Nervous system disorder (217) Hereditary disease (172) Neuromuscular disease (98) Syndromic disease (24) Human disease (14) Hereditary neurological disease (5) Congenital myasthenic syndrome (4) Hereditary neuromuscular disease (3) Disease of genetic or genomic mechanism (2)

Not yet recruiting 1

Gene therapy readiness check: new study maps virus immunity in muscle disease patients

Knowledge-focused Not yet recruiting

This study will test blood samples from 450 people aged 6 to 60 with inherited neuromuscular diseases to see if they have antibodies that could block gene therapy viruses (AAVs). The goal is to understand how common these antibodies are and which patients might be good candidates…

Phase: NA • Sponsor: Genethon • Aim: Knowledge-focused

Last updated Jun 27, 2026 13:04 UTC