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Ataluren open-label phase 2 study in infants and toddlers with nonsense mutation Duchenne muscular dystrophy
2020-000980-21Phase 2
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Jun 2021
- 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: Overall safety profile.
02
Drugs
ataluren
03
Conditions
Duchenne muscular dystrophy (DMD)
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