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Ataluren in Nonsense Mutation Methylmalonic Acidemia
NCT01141075Phase 2Terminated
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Jul 2010
- Primary completion
- Nov 2011
- Modality
- Small Molecules
- Countries
- 6
01
Overview
Phase 2, open-label, single-arm trial of ataluren in methylmalonic acidemia (MMA), sponsored by PTC Therapeutics, Inc. Primary endpoint: Plasma Methylmalonic Acid (MMacid) Levels at Day 29. Started Jul 2010; status: terminated.
02
Drugs
ataluren
03
Conditions
methylmalonic acidemia (MMA)
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