News & Blog

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

CIRM announces $7.4 million funding award for CRISPR/Cas9-mediated gene editing of hematopoietic stem and progenitor cells for Friedreich’s ataxia

Solid Biosciences Receives FDA Rare Pediatric Disease Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia

Lexeo Therapeutics Reports Third Quarter 2025 Financial Results and Operational Highlights

Lexeo Therapeutics Announces Progress in FDA Discussions for Accelerated Approval Pathway and Positive Interim Clinical Data for LX2006 in Friedreich Ataxia Cardiomyopathy

Larimar Therapeutics Announces Positive Data from Ongoing Long-term Open Label Study and Updates to Nomlabofusp Program for Friedreich’s Ataxia

Webinar Recording: Solid Biosciences September 2025 Update