New drug for rare congenital myasthenic syndromes
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study tests a new medicine called adimanebart for people with certain types of congenital myasthenic syndromes (CMS), a rare condition that causes muscle weakness. It aims to see if the drug can improve muscle strength and daily function.
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 take certain CMS medications (like albuterol or ephedrine), you've been on them for at least 6 months and can keep the same dose during the study
- For the second part of the study: you completed the first part of this trial (ARGX-119-2302)
- You do not have any other medical conditions that could make the study unsafe or affect results
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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