CONGENITAL MYASTHENIC SYNDROME
Clinical trials for CONGENITAL MYASTHENIC SYNDROME explained in plain language.
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Early trial tests stem cell particles for rare Muscle-Weakening disorder
Disease control Recruiting nowThis early-stage study is testing whether a solution containing particles from stem cells (called exosomes) can help people with congenital myasthenic syndrome, a rare genetic disorder that causes muscle weakness. The trial will enroll 20 participants to see if the treatment is s…
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Phase: PHASE1 • Sponsor: The Foundation for Orthopaedics and Regenerative Medicine • Aim: Disease control
Last updated Apr 01, 2026 14:43 UTC
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First human test for new Muscle-Weakness treatment
Disease control Recruiting nowThis is an early-stage study to check the safety and side effects of an experimental drug called ARGX-119 in adults with a rare genetic muscle disorder (DOK7-CMS). The study will also see how the body processes the drug and if it helps improve patients' strength and daily functio…
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Phase: PHASE1 • Sponsor: argenx • Aim: Disease control
Last updated Apr 01, 2026 14:43 UTC
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Scientists track rare muscle disease to unlock its secrets
Knowledge-focused Recruiting nowThis study aims to learn more about Congenital Myasthenic Syndrome (CMS), a rare genetic muscle-weakening disorder. Researchers will observe up to 100 participants with specific genetic types of CMS over several visits, tracking their symptoms, daily function, and quality of life…
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Sponsor: argenx • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:32 UTC