CONGENITAL MYASTHENIC SYNDROME
Clinical trials for CONGENITAL MYASTHENIC SYNDROME explained in plain language.
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New hope for rare muscle disease: ARGX-119 enters human testing
Disease control Recruiting nowThis early-stage trial tests a new biologic drug called ARGX-119 in 16 adults with a rare genetic muscle weakness condition called DOK7-congenital myasthenic syndrome (CMS). The study aims to see if the drug is safe and how the body processes it. Participants will receive either …
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Phase: PHASE1 • Sponsor: argenx • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Nasal spray made from stem cells could help rare muscle condition
Disease control Recruiting nowThis early-stage trial is testing a nasal spray made from stem cell exosomes in 20 people with congenital myasthenic syndrome, a rare genetic condition that causes muscle weakness. The goal is to see if the treatment is safe and can improve oxygen levels. Because it is a Phase 1 …
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Phase: PHASE1 • Sponsor: The Foundation for Orthopaedics and Regenerative Medicine • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Researchers track rare muscle disease to uncover its secrets
Knowledge-focused Recruiting nowThis study is for people with a rare genetic muscle condition called congenital myasthenic syndrome (CMS). It does not test any new drug or treatment. Instead, researchers will collect information from up to 100 participants over 4 visits to better understand how the disease affe…
Matched conditions: CONGENITAL MYASTHENIC SYNDROME
Sponsor: argenx • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC