News & Blog

Press Release: Graviton BioScience Announces Clearance of Investigational New Drug (IND) for GV101 for the Treatment of Friedreich’s Ataxia

Community Statement: Update from Biogen

Press Release: Cellares and Papillon Therapeutics Partner to Automate Manufacturing of PPL-001 for Patients With Friedreich’s Ataxia

Community Statement: Update from Biogen

Webinar Recording: PTC Therapeutics June 2026

Press Release: Larimar Therapeutics Reports Positive Open Label Data and Submission of First Module of Rolling BLA for Accelerated Approval of Nomlabofusp for Friedreich’s Ataxia

Webinar Recording: Lexeo Therapeutics May 2026

Press Release & Community Statement: Lexeo Therapeutics Announces Publication in JAMA Cardiology of Phase I/II Data for LX2006 in Friedreich Ataxia

Press Release: Update from Lexeo Therapeutics

Webinar Recording: Solid Biosciences May 2026

Community Letter: Update from PTC Therapeutics

Design Therapeutics Announces Four-Week IV Data from the RESTORE-FA Trial of DT-216P2 Demonstrating Clinical Improvements and Comprehensive Biomarker Activity in FA