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

MEMPHIS, Tenn. – Today, Le Bonheur 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 patients aged 4 through 5 years with a confirmed mutation in the DMD gene.

“It has taken teamwork from every department at Le Bonheur to get us to where we are today and treating out first patient with ELEVIDYS,” said Dr. Elena Caron and Dr. Daniel Guillien, Co-Directors of the Muscular Dystrophy Association Clinic /Neuromuscular clinic at Le Bonheur. Dr. Caron and Dr. Guillen are also associate and assistant professors of pediatric neurology at Le Bonheur and University of Tennessee Health Science Center. “From its approval in June to treating our patient today everyone has come together in an absolute collaboration to begin treating our Duchenne patients with ELEVIDYS. Today was a great day with the culmination of lot of work coming together for our patient.”

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 proteinby 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 . Additional information for patients is also available at www.SareptAssist.com.

 

About Le Bonheur Children’s 

Le Bonheur Children’s, based in Memphis, Tenn., provides expert care for children in more than 45 pediatric subspecialties, encompassing robust community programs, a pediatric research institute and regional outpatient centers in Jackson, Tenn., Tupelo, Miss., and Jonesboro, Ark.  Le Bonheur also features a 255-bed hospital in Memphis and a 21-bed satellite hospital within Jackson-Madison County General Hospital in Jackson, Tenn. As the primary pediatric teaching affiliate for the University of Tennessee Health Science Center, Le Bonheur trains more than 350 pediatricians and specialists each year. Nationally recognized, Le Bonheur has been named a U.S. News & World Report Best Children’s Hospital for 13 consecutive years and is a Magnet-designated facility, the ultimate credential for high-quality patient care and nursing excellence.  

For more information, please call (901) 287-6030 or visit lebonheur.org. Connect with us at , or on Instagram at. 

About ELEVIDYS

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.


Posted: 10/11/23