Navigating the Future of Gene Therapy in Neuromuscular Disorders: John Brandsema, MD

WATCH TIME: 4 minutes

"We need to be better at risk stratifying and thinking about who might need a different cocktail of medications to support them through the gene therapy process."

In 2023, the neuromuscular community celebrated another major milestone, as the FDA approved Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (Elevidys) as the first and only gene therapy for patients with Duchenne muscular dystrophy (DMD). Nearly a year later, the agency granted traditional approval to the one-time treatment, as well as expanded its indication to include patients aged 4 years and older with DMD who have a confirmed mutation in the DMD gene.

Gene therapy has represented a promising area of research for several subspecialites of neurology, as the advances in genetic testing have opened the door to understanding more about the genetic underpinnings of these disorders. For diseases like DMD and spinal muscular atrophy, gene therapy has already shown encouraging results, offering hope for improved motor function, longer survival, and better quality of life for patients. A hot topic in the field, the discussion around gene therapy will be raised several times at the upcoming Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, set to take place March 16-19 in Dallas, Texas.

Prior to the event, NeurologyLive® sat down with neuromuscular expert John Brandsema, MD, to give insight on the “Gene Therapy Updates” track he’s leading. Brandsema, a pediatric neurologist at Children’s Hospital of Philadelphia, provided comment on the current trends in gene therapy across various specialties, including insights from other disease areas. He spoke about the potential reality where multiple gene therapies are available for the same disorder and how to navigate treatment decisions. Furthermore, he detailed ways to optimize gene therapy delivery, risk stratification, and management of potential adverse events.