The US FDA has approved Elevidys, the first gene therapy to treat Duchenne muscular dystrophy (DMD) in children between the ages of four and five with confirmed mutations in the DMD gene. DMD is a genetic condition in which an absence of dystrophin – a protein that keeps muscle cells intact – causes weakness and wasting away of the body’s muscles. While most current treatments focus on management of symptoms, Elevidys is a recombinant gene therapy designed to provide the body with a gene that leads to production of a shortened form of the dystrophin protein. The therapy has been given accelerated approval and requires a clinical study to confirm its clinical benefit. Elevidys is produced by Sarepta Therapeutics.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy