PTC Therapeutics
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Key Inclusion Criteria: * mFARS ≥20 to ≤70 at Screening (4 weeks prior to Day 1) and Baseline (Day 1). * Must be ambulatory as defined by a E7 score of 4 or less on the USS at Screening and Baseline. * Documentation that participants reached maximum score on items E4, E5, and E3b on the USS of mFARS at Screening and Baseline. * FA diagnosis (homozygous for guanine-adenine-adenine \[GAA\] repeat expansion in intron-1 of the frataxin gene), confirmed and documented with GAA repeat length for both alleles by clinical genetic testing. * Difference in the mFARS score between Screening and Baseline…
Vatiquinone will be administered per dose and schedule specified in the arm.