News & Blog

The Oxford-Harrington Rare Disease Centre Announces Newly Funded Grant Award Programmes to Develop New Therapies for Friedreich’s Ataxia

2024 Baton Rouge Research Reception Recap

Design Therapeutics Announces First Quarter 2024 Financial Results and Highlights Upcoming Program Milestones

Meet the Community – Jason Drake

Astellas shares that US FDA cleared their IND with Fast Track designation for Phase 1 trial of their investigational gene therapy for the treatment of cardiomyopathy in FA

Lexeo Therapeutics Announces License Agreement to Accelerate Development of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy

Muscular Dystrophy Association and Friedreich’s Ataxia Research Alliance Announce Collaborative Research Grant Using Novel Gene Editing Technology to Address Root Cause of Friedreich’s Ataxia Disease

Lexeo Therapeutics Granted FDA Fast Track Designation for LX2006, an AAV-Based Gene Therapy Candidate for the Treatment of Friedreich’s Ataxia Cardiomyopathy

Meet the Community – Gabriel Queiroz Pereira & Fernanda Cenci Queiroz

rideATAXIA Gainesville 2024 Recap

Meet the Community – Joe Tyler

Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results