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Ataluren in nonsense mutation Duchenne muscular dystrophy
CTR20180881Phase 3Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Aug 2018
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing Ataluren with Placebo then ataluren (2 arms). Primary endpoints: Slope of change in 6-minute walk distance over 72 weeks during double-blind period and Comparison of early vs delayed ataluren initiation on functional outcomes. Started Aug 2018; status: completed.
02
Drugs
ataluren
03
Conditions
Duchenne muscular dystrophy (DMD)
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