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Friedreich's Ataxia
Biotech
FDA snubs PTC's Friedreich's ataxia hopeful with CRL
Despite missing its primary endpoint in a phase 3 study, PTC had hoped that another endpoint would help vatiquinone pass muster with the FDA.
Zoey Becker
Aug 19, 2025 10:55am
Lexeo locks in pivotal trial plan as gene therapy data accrue
Apr 7, 2025 9:37am
PTC bounces back with positive data on FA prospect vatiquinone
Oct 8, 2024 9:34am
Lexeo links gene therapy to improvements in rare heart disease
Jul 15, 2024 9:46am
FDA picks Denali, Neurogene and more for rare disease program
Jun 4, 2024 2:51pm
Larimar's Friedreich's ataxia program free at last from FDA hold
May 21, 2024 9:55am
