Sarepta Therapeutics Inc., a leader in precision genetic medicine for rare diseases, has announced ongoing regulatory review activities for their gene therapy, ELEVIDYS, aimed at treating Duchenne muscular dystrophy (DMD). While ELEVIDYS has received initial FDA approval in the U.S. and subsequent regulatory approvals in several other countries, it recently faced a setback in the European Union. The Committee for Medicinal Products for Human Use $(CHMP)$ issued a negative opinion on the conditional marketing authorization for the therapy. Despite this, Sarepta, in collaboration with its partner Roche, is committed to continuing discussions with the European Medicines Agency to explore potential pathways to bring ELEVIDYS to patients in the EU. The therapy has shown clinically meaningful improvements in motor function in various studies, and Sarepta remains focused on addressing outstanding safety questions with regulators.