Answers for Duchenne

Le Bonheur treats first patient in Tennessee with the first gene therapy for Duchenne muscular dystrophy 

Le Bonheur’s Neuroscience Institute recently treated the first patient in Tennessee 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 4 to 5 years old with a confirmed mutation in the DMD gene.  

Elena Caron, MD, co-director of the Muscular Dystrophy Association Clinic/ Neuromuscular Clinic

“It has taken teamwork from every department at Le Bonheur to get us to where we are today and treating our first patient with ELEVIDYS,” said Elena Caron, MD, and Daniel Guillen, MD, co-directors of the Muscular Dystrophy Association Clinic/ Neuromuscular Clinic at Le Bonheur. “From its approval in June to treating our first patient, everyone has come together in an absolute collaboration to begin treating our Duchenne patients with ELEVIDYS. It was the culmination of a lot of work coming together for our patient.”  

Daniel Guillen, MD, co-director of the Muscular Dystrophy Association Clinic/ Neuromuscular Clinic

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. No cure exists 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 to 30s.  

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.