Florida Nemours Children’s Hospital treats first patient with gene therapy

ORLANDO, Fla. — Nemours Children’s Hospital, Florida, has been recognized as an official treatment site for gene therapy to treat a rare muscular disease.

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ELEVIDYS from the Sarepta Therapeutics is now used to treat Duchenne Muscular Dystrophy.

Dr. Omar Abdul Hamid, a pediatric neurologist at Nemours Children’s Hospital, treated the first patient in Florida with this disease.

DMD is caused by a genetic variant that affects keeping muscle cells intact. According to the hospital, Duchenne primarily affects males and about 250,000 to 300,000 people worldwide.

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Symptoms show in childhood with muscle weakness and can interfere with the ability to walk, leading to a lower life expectancy.

The U.S. Food and Drug Administration approved the treatment as the first gene therapy for DMD. There is no current cure for the disease, but Nemours’ experts said they are hopeful it can halt disease progression.

“This innovative, new gene therapy will treat the cause of the disease,” Hamid said. “And is another tool for us as providers to help treat these patients; we are happy to be able to offer it to our patients who are candidates.”

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