June 20, 2024 (Los Angeles) — Neurologist Leigh Ramos-Platt, MD, Medical Director of the Muscular Dystrophy Association Neuromuscular Clinic at Children’s Hospital Los Angeles (CHLA), is available to comment on the FDA expanded approval of gene therapy for patients with Duchenne muscular dystrophy.
Ramos-Platt has treated six pediatric Duchenne muscular dystrophy (DMD) patients with Elevidys at Children’s Hospital Los Angeles with remarkable results. The new label expansion announced by the FDA Thursday is expected to enable CHLA to treat up to 100 more patients who were not previously eligible. “This is great news; a huge breakthrough for neuromuscular medicine,” says Ramos-Platt, who spent Thursday reaching out to patient families to alert them of the label expansion for gene therapy that was previously restricted to 4- and 5-year-old boys.
CHLA’s Neurological Institute operates one of the largest pediatric Duchenne programs in California.
“I am overwhelmed with joy,” Ramos-Platt said Thursday after news of the FDA approval broke. “In fact, I am calling families of our patients right now. I am thrilled that the miracles I have seen in six patients we have treated and the gene therapy will now be available for almost every one of our kids with Duchenne over the age of four with the confirmed mutation in the DMD gene.”
Ramos-Platt said there is a small number of patients who are ineligible because they have a contraindicated mutation that makes them ineligible for the treatment to fend off this life-threatening condition. “I am sad for them,” she says. “But we have many patients whose lives will be changed, and I am so happy for them.”
According to an FDA press release, “Duchenne muscular dystrophy is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body’s muscles. The disease occurs due to a defective gene that results in abnormalities in, or absence of, dystrophin, a protein that helps keep the body’s muscle cells intact.” The disease typically affects boys.
Media contact: Lorenzo Benet lbenet@chla.usc.edu 323-361-4823
