News & Blog

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

January/February 2026 – Advocacy Newsletter

Meet the Community: Josh Wooten

Meet the Community: Preston Lynch

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

Job Posting: Clinical Operations Manager

Meet the Community: Jamie Cusick

Meet the Community: Aly Bourbeau

December 2025 – Advocacy Newsletter

Meet the Community: Giovanna Boscolo Castilho Gonçalves