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Phase 2 safety, PK and PD study of ataluren in boys aged 2 to <5 years with nonsense mutation dystrophinopathy
2016-001764-11Phase 2
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Jan 2020
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of ataluren in Duchenne muscular dystrophy (DMD), sponsored by PTC Therapeutics, Inc. Primary endpoint: Safety of ataluren.
02
Drugs
ataluren
03
Conditions
Duchenne muscular dystrophy (DMD)
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