News & Blog

Webinar Recording: PTC Therapeutics Vatiquinone Program January 2025 Update

Janaury 2025 – Advocacy Newsletter

Larimar Therapeutics Announces Dosing of Adolescents in Nomlabofusp Pediatric Pharmacokinetic Run-In Study for Patients with Friedreich’s Ataxia

Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia

Press Release & Community Letter: Solid Biosciences Announces FDA IND Clearance for First-In-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich’s Ataxia

December 2024 – Advocacy Newsletter

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

Highlights of FA research from ICAR 2024

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