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Scientists Find Drug That May Help In Fight Against Duchenne Muscular Dystrophy

Posted Dec 12 2012 9:32pm

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

Now, scientists at UCLA have identified an additional drug — one that is already approved by the U.S. Food and Drug Administration and is being used in humans — that provides a powerful boost to the therapy now being tested in clinical trials. They hope that when used in combination, the drugs will provide a one-two punch that will overcome the genetic mutations that cause Duchenne muscular dystrophy (DMD), restore a missing protein needed for proper muscle function, and allow those affected by the disease to lead relatively normal lives.

The drug, called dantrolene, was found after researchers examined thousands of small molecules using a high-throughput molecular screening technique that allows them to scrutinize many molecules at the same time, said the study’s senior authors, Dr. Stanley Nelson, a UCLA professor of human genetics, and Carrie Miceli, a UCLA professor of microbiology, immunology and molecular genetics.


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