News & Blog

November 2024 – Advocacy Newsletter

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

Global Ataxia Advocacy Organizations Unite to Host Largest International Congress for Ataxia Research in London

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

September 2024 – Advocacy Newsletter

Congressional Resolution Passes to Designate September 25 as “National Ataxia Awareness Day”

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