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Long-term open-label ataluren safety and efficacy in nonsense mutation Duchenne muscular dystrophy (Study 049)
jRCT2041200087Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Jan 2021
- Modality
- Small Molecules
- Countries
- 2
01
Overview
Open-label, single-arm trial of ataluren in Duchenne muscular dystrophy (DMD), sponsored by PTC Therapeutics, Inc. Started Jan 2021; status: completed.
02
Drugs
ataluren
03
Conditions
Duchenne muscular dystrophy (DMD)
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