FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy.

The U.S. Food and Drug Administration (FDA) has expanded the approval of Elevidys, a gene therapy, for the treatment of Duchenne muscular dystrophy (DMD). The therapy is now approved for both ambulatory and non-ambulatory individuals aged 4 years and older with a confirmed mutation in the DMD gene. This decision was made based on the evidence of the therapy's clinical benefit, the urgent need for treatment, and the risks associated with the disease. Duchenne muscular dystrophy is a rare genetic condition that causes muscle weakness and wasting. Elevidys is a recombinant gene therapy that delivers a gene to produce a shortened protein that helps keep muscle cells intact. The most common side effects of Elevidys include vomiting, nausea, acute liver injury, fever, and low platelet count. The FDA granted approval to Sarepta Therapeutics Inc. for Elevidys. [Extracted from the article]