Nicklaus Children’s Expands Offering of Approved Gene Therapy for Duchenne Muscular Dystrophy Following FDA Decision

Published on: 09/18/2024

Nicklaus Children's Hospital is offering FDA-approved Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy developed for patients with Duchenne Muscular Dystrophy (DMD) who have a confirmed mutation in the DMD gene. Developed by Sarepta Therapeutics, ELEVIDYS was approved last year by the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD) in individuals ages 4 through 5 years old. In June of 2024, the FDA expanded approval of the drug to patients 4 years of age and older. The FDA granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients.

"We are excited to offer this emerging treatment to patients who have been eagerly awaiting the FDA's decision on expanding availability to additional age groups," said Dr. Migvis Monduy, medical director of the Neuromuscular and Movement Disorders Program at Nicklaus Children's Hospital. "We are hopeful that the treatment will delay or halt the progression of DMD for all eligible children, giving them the best chance for a better future."

Duchenne is a rare, genetic disease characterized by progressive muscle damage and weakness that occurs in approximately one in every 3,500-5,000 newborn males worldwide. It is caused by mutations in the dystrophin gene that lead to a lack of dystrophin protein. There is no cure for Duchenne, and the leading causes of death in individuals with Duchenne are respiratory or cardiac failure, which typically occurs when patients are in their mid-20s/30s. For more information and resources about Duchenne, please visit www.duchenne.com.

Nicklaus Children's is one of only three hospitals in Florida offering the treatment and was among the first in the nation to offer treatment to a 5-year-old boy. He is now able to jog and jump a year after receiving treatment and is progressing well. Jacob now looks forward to his physical therapy sessions and will be celebrating his 7 birthday soon.

This decision from the FDA allows for more people living with Duchenne than ever before to access the disease-modifying therapy with the potential to keep them living stronger for longer than the once certain trajectory of the disease. Nicklaus Children's is always at the forefront of pediatric medicine offering innovative treatments to children who need it the most. Nicklaus Children's is where your child matters most.