Nonsene Mutation Duchenne Muscular Dystrophy
Rare Diseases
1
Pipeline Programs
1
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1
Clinical Trials
0
Approved Products
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Preclinical
Phase 1
Phase 1/2
Phase 2
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Early DiscoveryClinical DevelopmentMarket
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1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
PTC TherapeuticsAtaluren
Clinical Trials (1)
Total enrollment: 6 patients across 1 trials
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Start: Dec 2021Est. completion: Aug 20236 patients
Phase 2Completed
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