News & Blog

Community Statement: Biogen announces BRAVE Phase 3 Pediatric Study of Omaveloxolone

April/May 2025 – Advocacy Newsletter

Webinar Recording: Solid Biosciences April 2025 Update

Press Release & Community Update: Lexeo Therapeutics Announces Positive Interim Phase 1/2 Data for LX2006 in Friedreich Ataxia Cardiomyopathy Supporting Advancement to Registrational Study

March 2025 – Advocacy Newsletter

Press Release & Community Update: Lexeo Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Operational Highlights

Larimar Therapeutics Provides Nomlabofusp Development Update and Reports Fourth Quarter and Full Year 2024 Financial Results

SKYCLARYS™ (omaveloxolone) Approval by Health Canada Ushers in a New Era for Friedreich’s Ataxia Treatment in Canada

Design Therapeutics Initiated Phase 1 Single Ascending Dose Trial of DT-216P2 in Healthy Volunteers

FA Community Statement from Biogen

Press Release & Community Statement: PTC Therapeutics Announces FDA Acceptance and Priority Review for Vatiquinone NDA for the Treatment of Children and Adults with Friedreich’s Ataxia

February 2025 – Advocacy Newsletter