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Open-label pharmacokinetic, safety, and efficacy study of vatiquinone in children with Friedreich ataxia under 7 years
2022-003265-38Phase 2
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Oct 2022
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of vatiquinone in Friedreich ataxia (FA), sponsored by PTC Therapeutics, Inc. Primary endpoint: Plasma Concentration of Vatiquinone.
02
Drugs
vatiquinone
03
Conditions
Friedreich ataxia (FA)
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