Sarepta Therapeutics' shares jumped 30% on June 21st after FDA expanded approval for its gene therapy Elevidys, treating Duchenne muscular dystrophy in patients 4 years and older.

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ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.