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New Treatment for Spinal Muscular Atrophy

Jerry Mendell, MD, FAAN

Research led by Jerry Mendell, MD has uncovered a one-time treatment for children with Spinal Muscular Atrophy–a cure for what is otherwise a fatal disease. And he is building on this treatment to develop similar therapies for patients with Duchenne and Limb-Girdle Muscular Dystrophy.

Dr. Mendell’s work involves gene therapy, a relatively new treatment option; the world saw its first gene therapy patient in 1990. Gene therapy shows promise for treating diseases for which there is no other cure.

Gene therapy requires three parts: a transgene to compensate for a faulty gene, a vector to deliver the transgene to a cell, and a promoter to turn that gene on in the right ways. In Dr. Mendell’s treatments, the vector is a harmless virus that “infects” cells with its DNA–DNA that was modified to include the new, healthy gene. Beyond muscular dystrophies, this kind of revolutionary therapy is being studied for the treatment of Alzheimer’s and Parkinson’s diseases, as well as ALS and many other neurological diseases.

The American Brain Foundation honored Dr. Mendell at our 2019 Commitment to Cures benefit event with the Cure One, Cure Many Award for his breakthrough research and for devoting his career to curing neuromuscular disorders. Dr. Mendell’s patients are living proof that when we cure one disease, we will cure many.

Learn more about diseases like Spinal Muscular Atrophy