Neuromuscular disease caused by qualitative or quantitative defects of dystrophin
MONDO:0016147Also known as: dystrophinopathy, qualitative or quantitative defects of dystrophin
5 clinical trials for this condition and its sub-types, 4 tagged with Neuromuscular disease caused by qualitative or quantitative defects of dystrophin itself.
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Browse by category →Sub-types of Neuromuscular disease caused by qualitative or quantitative defects of dystrophin
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Dilated cardiomyopathy 3B 1 trial
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New drug aims to save hearts in duchenne muscular dystrophy
Disease control CompletedThis study tested an oral drug called ifetroban in 46 males aged 7 and older with Duchenne muscular dystrophy (DMD). The goal was to see if the drug is safe and can help prevent or treat heart problems, which are a leading cause of death in DMD. Participants received either ifetr…
Phase 2 • Sponsor: Cumberland Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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Experimental drug ataluren tested for safety in duchenne MD patients
Disease control CompletedThis study tested the safety of a drug called ataluren (also known as Translarna) in 270 people with Duchenne muscular dystrophy caused by a specific type of genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies. The goal was to monitor s…
Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC