News & Blog

PTC Therapeutics Announces Vatiquinone NDA Submission to FDA for the Treatment of Children and Adults Living with Friedreich’s Ataxia

Larimar Therapeutics Announces Positive Initial Data from Ongoing Long-term Open Label Extension Study & Progress Across Nomlabofusp Program for Friedreich’s Ataxia

Larimar Therapeutics Presents Additional Data from Phase 1 Studies and Phase 2 Dose Exploration Study Supporting the Nomlabofusp Clinical Program at ICAR 2024

Lexeo Therapeutics Announces Complete Enrollment of LX2006 SUNRISE-FA Phase 1/2 trial and Alignment with FDA on an Accelerated Approval Pathway

Larimar Therapeutics Announces Three Poster Presentations at the 2024 International Congress for Ataxia Research (ICAR) on November 12–15, 2024

Capsida Biotherapeutics to Present New Preclinical Data for Friedreich’s Ataxia Next-Generation Gene Therapy to Target CNS, Cardiac Tissues and Sensory Regions

Papillon Therapeutics Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich’s Ataxia

PTC Therapeutics Announces Positive Results from Long-Term Treatment Studies and Updates on Regulatory Progress for Vatiquinone Friedreich Ataxia Program

Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich’s Ataxia

Stealth BioTherapeutics Awarded Grant from Friedreich’s Ataxia Research Alliance and FARA Ireland to Develop Investigational Mitochondrial Therapeutic SBT-589 for Friedreich’s Ataxia

Larimar Therapeutics Reports Several Updates on Nomlabofusp FA Program in Q2 2024 Operating and Financial Results

LEXEO Therapeutics Announces Positive Interim Phase 1/2 Clinical Data of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy