Providers at Nemours Children’s Hospital in Orlando treated a DMD patient with Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), which the FDA approved June 22 for 4- and 5-year-olds with a DMD gene mutation. The treatment costs $3.2 million.
Elevidys is the first and only approved gene therapy for the rare genetic condition that weakens the body’s muscles. DMD mostly affects males, and between 9,000 and 12,000 people in the U.S. are diagnosed with it, according to Nemours Children’s Hospital.
“Until now, patients have only had treatments to manage their symptoms,” the hospital said in a Sept. 26 news release.
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