Pediatric experts from Le Bonheur Children’s Hospital and the University of Tennessee Health Science Center will present their research at the annually held National Conference & Exhibition of the American Academy of Pediatrics (AAP) held October 20-24, 2023 in Anaheim, CA.
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.
Le Bonheur Children's Hospital is one of the nation's best children's hospitals, featuring expert physicians and staff, state-of-the-art technology and family-friendly resources. We are also part of the Methodist Le Bonheur Healthcare group of hospitals.