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Results for “Pontocerebellar Hypoplasia Type 6 (PCH6)”

2 trials

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Showing 2 of 2 results

Large-scale testing (Phase 3)Study completedNCT05218655
What this trial is testing

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Who this might be right for
Inherited Mitochondrial Disease
PTC Therapeutics 101
Large-scale testing (Phase 3)Ended earlyNCT04378075
What this trial is testing

Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Who this might be right for
Mitochondrial DiseasesDrug Resistant EpilepsyLeigh Disease+5 more
PTC Therapeutics 68