Congenital myopathy
MONDO:0019952Also known as: congenital myopathy, Batten Turner congenital myopathy, myopathy congenital
76 clinical trials for this condition and its sub-types, 8 tagged with Congenital myopathy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Congenital myopathy
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Congenital structural myopathy 5 trials · 62 incl. sub-types
6 sub-types
- Inborn mitochondrial myopathy 18 trials · 45 incl. sub-types Sub-types →
- Nemaline myopathy 13 trials Sub-types →
- Myofibrillar myopathy 1 trial · 3 incl. sub-types Sub-types →
- Congenital fiber-type disproportion myopathy 1 trial Sub-types →
- Autosomal dominant centronuclear myopathy 0 trials
- Autosomal dominant nebulin-related myopathy 0 trials
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RYR1-related myopathy 5 trials · 6 incl. sub-types
5 sub-types
- Central core myopathy 2 trials
- King-Denborough syndrome 0 trials
- Congenital multicore myopathy with external ophthalmoplegia 0 trials
- Congenital myopathy with myasthenic-like onset 0 trials
- Rhabdomyolysis-myalgia syndrome 0 trials
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Centronuclear myopathy 2 trials · 5 incl. sub-types
5 sub-types
- X-linked myotubular myopathy 4 trials Sub-types →
- Autosomal dominant centronuclear myopathy 0 trials
- Autosomal recessive centronuclear myopathy 0 trials Sub-types →
- Congenital myopathy with internal nuclei and atypical cores 0 trials
- Myopathy, centronuclear, 6, with fiber-type disproportion 0 trials
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TTN-related myopathy 2 trials · 4 incl. sub-types
2 sub-types
- Autosomal recessive titinopathy 0 trials · 2 incl. sub-types Sub-types →
- Autosomal dominant titinopathy 0 trials Sub-types →
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TPM2-related myopathy 1 trial · 3 incl. sub-types
2 sub-types
- Central core myopathy 2 trials
- Congenital myopathy 23 0 trials
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SELENON-related myopathy 1 trial
1 sub-type
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TPM3-related myopathy 1 trial
3 sub-types
- Cap myopathy 0 trials
- Congenital myopathy 4A, autosomal dominant 0 trials
- Congenital myopathy 4B, autosomal recessive 0 trials
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TOR1AIP1-related myopathy 0 trials · 1 incl. sub-types
1 sub-type
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Bailey-Bloch congenital myopathy 0 trials
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Batten-Turner congenital myopathy 0 trials
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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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Compton-North congenital myopathy 0 trials
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MEGF10-related myopathy 0 trials
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MYH7-related skeletal myopathy 0 trials
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1 sub-type
- Congenital myasthenic syndrome 16 0 trials
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4 sub-types
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Alpha-actinopathy 0 trials
4 sub-types
- Cap myopathy 0 trials
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Progressive scapulohumeroperoneal distal myopathy 0 trials
- Zebra body myopathy 0 trials
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Benign Samaritan congenital myopathy 0 trials
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Congenital myopathy 11 0 trials
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Congenital myopathy 15 0 trials
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Congenital myopathy 18 0 trials
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Congenital myopathy 20 0 trials
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Congenital myopathy 22A, classic 0 trials
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Congenital myopathy 25 0 trials
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Congenital myopathy 26 0 trials
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Congenital myopathy 27 0 trials
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Cylindrical spirals myopathy 0 trials
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Fingerprint body myopathy 0 trials
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Hyaline body myopathy 0 trials
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Reducing body myopathy 0 trials
2 sub-types
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Tubular aggregate myopathy 0 trials
2 sub-types
- Myopathy, tubular aggregate, 1 0 trials
- Myopathy, tubular aggregate, 2 0 trials
Most studied deeper sub-types
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New drug aims to tame hard-to-control seizures in rare mitochondrial disorders
Disease control Stopped earlyThis study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …
Phase 2/3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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New drug OMS721 tested for rare kidney conditions
Disease control Stopped earlyThis study tested a drug called OMS721 in 31 people with certain kidney diseases, including IgA nephropathy and lupus nephritis. The main goal was to check if the drug is safe and if it reduces protein in the urine, a sign of kidney damage. The study was stopped early, so results…
Phase 2 • Sponsor: Omeros Corporation • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC