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Congenital myasthenic syndrome 10

MONDO:0009690

Any congenital myasthenic syndrome in which the cause of the disease is a mutation in the DOK7 gene.

Also known as: CMS10, DOK7 congenital myasthenic syndrome, congenital muscular dystrophy merosin-positive, congenital myasthenic syndrome 10, congenital myasthenic syndrome caused by mutation in DOK7, congenital myasthenic syndrome type 10, myasthenic syndrome, congenital, type 10, CMS Ib

7 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Nervous system disorder (231) Musculoskeletal system disorder (207) Hereditary disease (176) Neuromuscular disease (106) Muscular dystrophy (72) Muscle tissue disorder (56) Syndromic disease (25) Human disease (14) Myopathy (14)
Trials to join now! 6 Not yet finished but already full! 1
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  • New hope for rare muscle disease: ARGX-119 enters human testing

    Disease control Ongoing

    This 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 Jul 26, 2026 00:00 UTC

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