This Science is Personal: Husband and Wife Research Team has Son with the Disease

Drugs are currently being tested that show promise in treating patients with Duchenne muscular dystrophy (DMD), an inherited disease that affects about one in 3,600 boys and results in muscle degeneration and, eventually, death.

Now, Drs. Stanley Nelson, Carrie Miceli, and their team of scientists at the Eli & Edythe Broad Center of Regenerative and Stem Cell Research at UCLA have found a drug, already approved by the U.S. Food & Drug Administration and being used in humans, that provides a powerful boost to the therapy currently being tested in clinical trials. They hope this one-two punch used in combination will overcome the genetic mutations that cause DMD, restore a missing protein needed for proper muscle function and allow those affected by the disease to lead relatively normal lives.

The study appears Dec. 12, 2012, in the peer-reviewed journal Science Translational Medicine.

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