CONGENITAL MYASTHENIC SYNDROME
Clinical trials for CONGENITAL MYASTHENIC SYNDROME explained in plain language.
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Could stem cell particles help rare muscle weakness disorder?
Disease control Recruiting nowThis early-phase trial tests whether a solution made from stem cell exosomes can help people with congenital myasthenic syndrome, a rare genetic condition causing muscle weakness. About 20 participants will receive the treatment and be monitored for safety and oxygen levels. The …
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Phase: PHASE1 • Sponsor: The Foundation for Orthopaedics and Regenerative Medicine • Aim: Disease control
Last updated May 17, 2026 06:50 UTC
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New hope for rare muscle disease: experimental drug ARGX-119 enters human testing
Disease control Recruiting nowThis early-stage study tests a new medicine called ARGX-119 in 16 adults with a rare genetic condition (DOK7-CMS) that causes muscle weakness. The main goal is to check if the drug is safe and how the body handles it. Participants will receive either the drug or a placebo, and la…
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Phase: PHASE1 • Sponsor: argenx • Aim: Disease control
Last updated May 17, 2026 06:50 UTC
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New study tracks rare muscle-weakening disease to uncover its secrets
Knowledge-focused Recruiting nowThis study is for people aged 2 and older with certain rare types of congenital myasthenic syndrome (CMS) caused by specific gene changes. Researchers will collect information from up to 4 visits to track symptoms, medication use, and quality of life. The goal is to better unders…
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Sponsor: argenx • Aim: Knowledge-focused
Last updated May 17, 2026 07:02 UTC