Congenital myasthenic syndrome
MONDO:0018940Congenital myasthenic syndrome (CMS) is a group of genetic disorders of impaired neuromuscular transmission at the motor endplate characterized by fatigable muscle weakness.
Also known as: CMS, Congenital Myasthenic Syndromes, myasthenic syndrome, congenital, congenital MG, congenital myasthenia, erb-Goldflam syndrome, familial limb-girdle myasthenia, myasthenia gravis congenital
5 clinical trials for this condition and its sub-types.
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Broader categories
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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 …
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 …
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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New study tracks rare muscle disorders to improve future care
Knowledge-focused Recruiting nowThis study follows 75 people aged 6 months or older with congenital myasthenic syndromes (CMS), a group of inherited disorders that weaken communication between nerves and muscles. Over up to 7 visits in 5 years, researchers will use physical exams, heart and lung tests, MRIs, an…
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) • Aim: Knowledge-focused
Last updated Jun 28, 2026 00:00 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…
Sponsor: argenx • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC