UHealth and Holtz Children’s Hospital physicians are exploring a new gene therapy called ELEVIDYS to treat older Duchenne muscular dystrophy (DMD) patients. The therapy delivers a functional micro-gene to muscle cells, potentially halting DMD progression and, in some cases, possibly reversing it, said Dr. Roberto Lopez-Alberola, an associate professor of neurology and pediatrics and chief of pediatric neurology at the University of Miami Miller School of Medicine and a neurologist at Holtz Children’s Hospital at Jackson Memorial Medical Center. ELEVIDYS received accelerated FDA approval in 2023, but the therapy could only be administered to children ages 4 and 5. In June 2024, the FDA expanded access to patients older than 4. “I have been working in neurogenetics for 30 years, and it’s amazing to see the dream of gene therapy being realized,” Dr. Lopez-Alberola said. “It’s a wonderful opportunity to treat these patients and see the impact it’s having.” DMD is a childhood muscular disorder driven by mutations in dystrophin, which is a gene that protects muscle cells from damage. Without the gene, children experience muscle weakness that progressively limits walking, breathing, blood flow and other functions. ELEVIDYS uses a harmless virus called AAVrh74 to deliver therapeutic DNA to muscle cells. However, viruses have capacity limits when being introduced to the body, Dr. Lopez-Alberola explained. To overcome the size problem, ELEVIDYS uses a micro-dystrophin that can both fit inside the virus and, hopefully, provide therapeutic benefit. Clinical trials have shown the drug can improve motor function, particularly in younger children. “The worst-case scenario is that some patients do not regain any function,” he said. “However, we may slow the disease’s progression. That alone would be transformational.” Dr. Lopez-Alberola said he hopes that the FDA will continue to extend the ELEVIDYS therapy age eligibility to include infants so they can be screened and treated in their first weeks of life. “Right now, we can’t treat until patients become symptomatic,” he said. “By then, some muscle has already been irreversibly lost. It would be much better if we could circumvent the natural history of the disease and prevent that from happening.” Related Posts:Mount Sinai cancer center to probe patient experiencesNew medical approach guides Nicklaus Hospital cancer…Mount Sinai cancer center clears Miami Beach hurdleUHealth Doral opening piece by piece through 2026County pushes for mobile stroke unitThe post UHealth, Holtz Children’s exploring gene therapy appeared first on Miami Today.