Study of adimanebart for congenital myasthenic syndromes
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new medicine, adimanebart, for people with certain types of congenital myasthenic syndrome (CMS), a condition that causes muscle weakness. The study aims to see if the medicine improves muscle strength and daily function, and it includes both teenagers and adults.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 12 years old or older.
- You have a confirmed diagnosis of CMS caused by changes in the DOK7, MUSK, AGRN, or LRP4 genes.
- If you are currently taking certain CMS medications (like albuterol, salbutamol, or ephedrine), you must have been on them for at least 6 months and be willing to stay on the same dose during the study, unless your doctor changes it.
- You have completed the active treatment part of a previous study (ARGX-119-2302) to join the long-term follow-up portion.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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