Drug is first gene therapy for Duchenne muscular dystrophy

Last week, the US Food and Drug Administration granted accelerated approval of a new drug to treat ambulatory pediatric patients aged four to five years with Duchenne muscular dystrophy.

It is the first gene therapy approved to treat this debilitating and fatal disease found almost exclusively in boys.

“…there is clearly something happening that is new and very promising.”

Emma Ciafaloni is a neuromuscular neurologist with the University of Rochester Medical Center (URMC) neurology department and Golisano Children’s Hospital, and director of the UR Medicine Duchenne Muscular Dystrophy Clinic, which treats boys with Duchenne muscular dystrophy (DMD) from across upstate New York.

Ciafaloni has been involved in DMD clinical research for decades and URMC was one of first three sites in the nation to start dosing patients in the phase 3 clinical trial for the new gene therapy. The study, called EMBARK, has since expanded to additional sites in North America, Europe, and Asia. Ciafaloni also served as chair of the independent Data Safety and Monitoring Board for the early phase clinical trials of the therapy. The new drug—delandistrogene moxeparvovec-rokl—is being developed by Sarepta Therapeutics and marketed under the name ELEVIDYS.

Here, Ciafaloni describes the drug, why the FDA chose to limit its use, and what this means for patients and their families:

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