The treatment from Sarepta Therapeutics was approved Thursday for children ages 4 and 5 with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes early death.

Doctors say it is the biggest advancement in years. "The new gene therapy for Duchenne muscular dystrophy is a major breakthrough," said Dr. Jerry Mendell, with Nationwide Children's Hospital.

First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids ... Updated: 4:28 AM EDT Jun 23, 2023 Editorial Standards ⓘ By MATTHEW PERRONE, AP Health Writer ...