Nicklaus Children’s Hospital Now Administering the First FDA-Approved Gene Therapy for Duchenne Muscular Dystrophy

Published on: 11/1/2023

Nicklaus Children’s Hospital announced it has treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy. Developed by Sarepta Therapeutics, ELEVIDYS is approved by the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD) in ambulatory pediatric patients ages 4 through 5 years with a confirmed mutation in the DMD gene. Nicklaus Children’s is one of the first in the nation and one of only three hospitals in Florida offering this new treatment.

Jacob, who just turned 6 and resides in Miami’s Little Havana neighborhood was treated on October 12.

“At Nicklaus Children’s we always seek the latest, emerging treatments to support children with complex disorders,” said Dr. Migvis Monduy, medical director of the neuromuscular and movement disorders programs. “It is our hope this treatment will delay or halt the progression of DMD for eligible children, offering them brighter tomorrows. Our use of emerging treatments is one more reason why Nicklaus Children’s is the hospital where children matter most.”

Jacob’s mother, Erika Escobar, said, “When doctors first gave me Jacob's diagnosis, the news made me especially sad because just a year earlier, I lost my only daughter to lupus in Honduras. I was afraid of losing him too. After processing everything and with the support of doctors and family, I looked at things differently. Being the first patient in South Florida to receive this treatment and for everything to be a success has given me hope for his 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. More information and resources about Duchenne can be found at https://www.duchenne.com/.

ELEVIDYS addresses the root genetic cause of Duchenne—mutations in the dystrophin gene that result in the lack of dystrophin protein—by delivering a gene that codes for a shortened form of dystrophin to muscle cells, known as ELEVIDYS-dystrophin. The therapy’s accelerated approval is based on an increase in ELEVIDYS-dystrophin protein expression in skeletal muscle cells.

For further information, please see the full Prescribing Information. Additional information for patients is also available at www.SareptAssist.com.

ELEVIDYS is a trademark of Sarepta Therapeutics, Inc. registered in the U.S. Patent and Trademark Office and may be registered in various other jurisdictions.  

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