CONGENITAL MYASTHENIC SYNDROME
Clinical trials for CONGENITAL MYASTHENIC SYNDROME explained in plain language.
Never miss a new study
Get alerted when new CONGENITAL MYASTHENIC SYNDROME trials appear
Sign up with your email to follow new studies for CONGENITAL MYASTHENIC SYNDROME, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
Could stem cell particles help treat rare muscle weakness disorder?
Disease control Recruiting nowThis early-phase trial tests whether a solution made from stem cell particles (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 …
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Phase: PHASE1 • Sponsor: The Foundation for Orthopaedics and Regenerative Medicine • Aim: Disease control
Last updated May 26, 2026 09:35 UTC
-
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 18, 2026 11:58 UTC
-
New study tracks rare muscle disease to uncover its secrets
Knowledge-focused Recruiting nowThis study is for people aged 2 and older with certain rare genetic forms of congenital myasthenic syndrome (CMS). It does not test a new treatment. Instead, researchers will collect information from up to 4 visits to learn how the disease changes over time and affects daily life…
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Sponsor: argenx • Aim: Knowledge-focused
Last updated May 26, 2026 09:56 UTC