News & Blog
News & Blog
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
Larimar Therapeutics Selected by FDA to Participate in START Pilot Program for Nomlabofusp in Friedreich’s Ataxia
Larimar Therapeutics Announces FDA has Removed Partial Clinical Hold for Nomlabofusp Program in Friedreich’s Ataxia
Food and Drug Administration (FDA) removed partial clinical hold following review of Phase 2 dose exploration study data Ongoing open l...