Here’s a New Treatment for Muscular Dystrophy

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In a statement released Wednesday, Sarepta Therapeutics (NASDAQ:SRPT) said its new treatment for Duchenne muscular dystrophy helped patients walk farther, pushing the company’s stock price up 165 percent to $39.67. Shares initially rose as high as $43.12, the largest intraday increase for the stock since June 1997.

The Cambridge, Massachusetts-based biotechnology company said the experimental drug, eteplirsen, met the primary goal of the mid-stage study; four patients who took 50 milligrams of the drug once a week for 48 weeks were able to walk 89 meters, or 292 feet, further in a six minute test than they were before beginning the trial. The four patients who made up the trial group also showed a remarkable improvement over patients who took a placebo for half of the study and the medication for the remaining 24 weeks.

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Eteplirsen helps Duchenne muscular dystrophy patients produce a missing protein and regain muscle strength by repairing a mutated gene, according to the companies statement. Symptoms of the disease, which usually appear in male children before the age six, include muscle loss in the legs, pelvis, and eventually the whole body. Female children are almost never diagnosed with the disease because it is caused by a defective gene on the X chromosome.

The results “may lead to first-ever opportunities to target serious and life-threatening rare conditions at the origin of disease,” said Sarepta Chief Executive Officer Chris Garabedian in the statement.

The positive results of the trial overshadowed an announcement that the U.S. Department of Defense planned to drop the Ebola portion of its contract for hemorrhagic fever virus therapeutics with Sarepta because of budget cuts.

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