Phase 1/2 Findings Highlight RGX-202's Safety, Impact on Key Duchenne Biomarkers
RGX-202, a gene therapy for Duchenne muscular dystrophy, was well tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.
United States Aravindhan Veerapandiyan Olivier Danos Manufacturing Innovation Center World Muscle Society International Congress
Source: ajmc.com