Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy
XTalks
JUNE 28, 2023
DMD is caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells. The FDA approval came after several delays and a narrow advisory committee vote of eight to six in favor of the gene therapy’s risk-benefit profile. It is injected into a patient’s muscle tissue.
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