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Phase 2 study of ataluren for nonsense mutation methylmalonic acidemia
2009-016654-41Phase 2Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Jan 2010
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, single-arm trial of ataluren in methylmalonic acidemia (MMA), sponsored by PTC Therapeutics, Inc. Primary endpoint: Change in plasma methylmalonic acid levels during 4 weeks of ataluren treatment. Started Jan 2010; status: completed.
02
Drugs
ataluren
03
Conditions
methylmalonic acidemia (MMA)
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