News & Blog

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

Job Posting: Development Manager (Major Gifts)

Letter to the FA Community from Astellas Pharma

Webinar Recording: PTC Therapeutics Vatiquinone Program January 2025 Update

Janaury 2025 – Advocacy Newsletter

Larimar Therapeutics Announces Dosing of Adolescents in Nomlabofusp Pediatric Pharmacokinetic Run-In Study for Patients with Friedreich’s Ataxia