Congenital muscular dystrophy
MONDO:0019950A muscular dystrophy that is characterized by diminished muscle tone (hypotonia), progressive muscle weakness and degeneration (atrophy), abnormally fixed joints, spinal rigidity, and delays in reaching motor milestones such as sitting or standing unassisted.
Also known as: CMD, MDC, congenital MD
14 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 OngoingThis 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 1 • Sponsor: argenx • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC
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Desperate hope: stem cells tested on one child with rare muscle disease
Disease control Expanded access (ended)This trial gives a single child with a rare form of congenital muscular dystrophy access to their own banked stem cells. The cells are given through 14 IV infusions to see if they are safe and can help control the disease. Because it involves only one patient, the results will be…
Sponsor: Hope Biosciences Research Foundation • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC