Clinical Trial Update: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome [Phase 2]

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Summary

This document summarizes the Phase 2 long-term safety and efficacy results of EPI-743 in children with Leigh syndrome.

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Diseases / indications

• Leigh Syndrome

Drugs mentioned

• EPI-743

Snippet

Sponsor: PTC Therapeutics | Conditions: Leigh Syndrome | Phase 2 | Status: COMPLETED