News & Blog

February 2023 – Advocacy Newsletter

A Gene-Edited Cell Therapy for an Incurable Disease: Researchers Receive USD 4.8M Grant to Treat Friedreich’s Ataxia

Dr. Stepanova Presented with $20,000 Research Grant from Uplifting Athletes

Prime Medicine announces recent progress and highlights 2023 strategic priorities

Hereditary Ataxias Added as Eligible Condition for CDMRP Funding!

Design Therapeutics Reports Positive Data from Single-Ascending Dose Trial of DT-216 for the Treatment of Friedreich Ataxia and Portfolio Progress

Strong Partnership Funds the 2022 Ataxian Athlete Initiative (AAI) for People with Ataxia

Design Therapeutics Completes Dosing in First Patient Cohort of Phase 1 Trial of DT-216 GeneTAC™ Molecule for the Treatment of Friedreich Ataxia

Stealth BioTherapeutics Receives Orphan Drug Designation from FDA for Elamipretide for the Treatment of Friedreich’s Ataxia

Design Therapeutics Announces FDA Clearance of Investigational New Drug Application for First GeneTAC™ Molecule for Friedreich Ataxia

Lexeo Announces FDA Clearance of IND Application for LX2006 for Friedreich’s Ataxia Cardiomyopathy

Larimar Therapeutics Provides Update on CTI-1601 Clinical Program