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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, 1 tagged with Congenital muscular dystrophy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Congenital muscular dystrophy
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Congenital myasthenic syndrome 10 2 trials
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Muscular dystrophy-dystroglycanopathy 0 trials · 1 incl. sub-types
7 sub-types
- Muscular dystrophy-dystroglycanopathy, type A 1 trial Sub-types →
- Muscular dystrophy-dystroglycanopathy, type C 0 trials · 1 incl. sub-types Sub-types →
- DPM3-congenital disorder of glycosylation 0 trials Sub-types →
- Congenital muscular dystrophy with intellectual disability 0 trials
- Congenital muscular dystrophy with intellectual disability and severe epilepsy 0 trials
- Muscle-eye-brain disease with bilateral multicystic leucodystrophy 0 trials
- Muscular dystrophy-dystroglycanopathy, type B 0 trials Sub-types →
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Rigid spine syndrome 0 trials · 1 incl. sub-types
1 sub-type
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Bethlem myopathy 0 trials
4 sub-types
- Bethlem myopathy 1A 0 trials
- Bethlem myopathy 1B 0 trials
- Bethlem myopathy 1C 0 trials
- Bethlem myopathy 2 0 trials
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1 sub-type
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4 sub-types
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1 sub-type
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Congenital muscular dystrophy 1B 0 trials
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Congenital myopathy, Paradas type 0 trials
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Muscle-eye-brain disease 0 trials
9 sub-types
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A, 4 0 trials
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A1 0 trials
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A14 0 trials
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A2 0 trials
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A3 0 trials
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A5 0 trials
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A6 0 trials
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type a, 10 0 trials
- Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type a, 11 0 trials
Most studied deeper sub-types
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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