News & Blog

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

Press release and community statement: Lexeo Therapeutics Reports First Quarter 2026 Financial Results and Operational Highlights

Community Statement: Update from Biogen

Community Statement: Update from Biogen

Webinar Recording: Larimar Therapeutics March 2026 Update

Press release and community statement: Lexeo Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights

Larimar Therapeutics Announces FDA Breakthrough Therapy Designation for Nomlabofusp in FA and Reiterates Planned BLA Submission in June 2026

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results

The Oxford-Harrington Rare Disease Centre Advances Discovery of Therapies for Friedreich’s Ataxia (FA)

Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

Solid Biosciences Doses First Participant in First-in-Class Phase 1b FALCON Trial Evaluating SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

Community Statement: 2025 Update from Biogen