Newswise — After previously approving its use for a limited population, the U.S. Food and Drug Administration has now approved Elevidys, a gene therapy designed to treat Duchenne muscular dystrophy (DMD), for a broader range of child patients.
Leigh Maria Ramos-Platt, MD, Medical Director of the Neuromuscular Disorders Center at Children’s Hospital Los Angeles, has previously treated 6 patients with DMD at CHLA using Elevidys and has seen excellent results. She is thrilled at the announcement of the FDA’s expanded approval. “This is great news; a breakthrough for the practice of neuromuscular medicine,” Dr.
Ramos-Platt says. The Neurological Institute at CHLA operates one of California’s largest pediatric DMD programs. Dr.
Ramos-Platt explains that the FDA’s newly expanded approval is expected to allow CHLA to treat many more patients with Elevidys—patients who were not eligible before. Having seen the benefits of Elevidys, Dr. Ramos-Platt is eager to be able to bring the gene therapy to more children with DMD.
“The consensus is that it's better than any alternative out there currently, even though there are likely to be better treatments in the future,” she explains. A genetic disorder leads to progressive degeneration DMD is a genetic condition in which a defective gene causes either abnormalities in, or the absence of a protein called dystrophin, which helps muscle cells to remain intact. This leads to muscle degeneration, which worsens over time.
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