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REGENXBIO Inc. (NASDAQ: RGNX) today announced Chief Medical Officer, Steve Pakola, M.D., will present at the International Congress of the World Muscle Society taking place in Vienna, Austria, October 7-11, 2025.
The podium presentation will share new analysis of previously presented 12-month functional data from the Phase I/II trial of RGX-202, including individual patient improvement on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model from the Collaborative Trajectory Analysis Project. As reported, RGX-202 demonstrated a favorable safety profile with no serious adverse events or adverse events of special interest observed in the Phase I/II study. Pivotal dose participants exceeded baseline-matched external natural history controls on all functional measures.
Using multiple models of natural history disease progression, these results further demonstrate the potential of RGX-202 to serve as a differentiated, best-in-class gene therapy for the treatment of Duchenne muscular dystrophy.
Posted In: RGNX
