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Dr. Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research (CBER) at the FDA, was our keynote speaker. CBER oversees the regulation and approval of all cell and gene therapies, including Sarepta’s Elevidys for 4-5-year-olds with Duchenne.
You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.
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