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Open-Label Long-Term Safety Study of Vatiquinone in Inherited Mitochondrial Disease
jRCT2011230045Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Oct 2023
- Modality
- Small Molecules
01
Overview
Open-label, single-arm trial of vatiquinone in Mitochondriopathy, Leigh syndrome, Alpers disease and 3 more, sponsored by PTC Therapeutics, Inc. Started Oct 2023; status: completed.
02
Drugs
vatiquinone
03
Conditions
MitochondriopathyLeigh syndromeAlpers diseasemitochondrial encephalomyopathiesmyoclonic epilepsy with ragged-red fibers (MERRF)
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