Clinical Trial Update: A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) [Phase 2]

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Summary

Phase 2 study evaluating dystrophin level changes in participants with nonsense mutation Duchenne muscular dystrophy completed; results posted in 2022.

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Diseases / indications

• Duchenne Muscular Dystrophy

Drugs mentioned

• PTC124/Nonsense mutation Duchenne muscular dystrophy therapy (drisapersen? (note: actual generic used for PTC Therapeutics with nonsense mutations is ataluren? If unspecified, flag as null))

Snippet

Sponsor: PTC Therapeutics | Conditions: Duchenne Muscular Dystrophy | Phase 2 | Status: COMPLETED | Results posted: 2022-04-05