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Promising early results for gene therapy for Duchenne muscular dystrophy

Researchers are reporting dramatic, if preliminary, results for a gene therapy treatment for an inherited muscle wasting disease. Three young boys with Duchenne muscular dystrophy who received gene therapy are now making at least 38% the normal level of dystrophin, a protein that is defective or missing in the disease, Forbes reports. In a video, a boy who could only climb one stair at a time quickly scales the same stairs after treatment, according to an investor presentation from the company Sarepta Therapeutics in Cambridge, Massachusetts. Two other companies are testing a similar treatment and one patient in these trials is reportedly doing well too, notes blogger Derek Lowe at the In the Pipeline blog, which is published by AAAS.

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