PUBLIC RELEASE DATE: 7-May-2014 Contact: Sally Stewart sally.stewart@cshs.org 310-248-6566 Cedars-Sinai Medical Center LOS ANGELES (May 7, 2014) Cedars-Sinai Heart Institute researchers have found that a commonly prescribed drug restores blood flow to oxygen-starved muscles of boys with Duchenne muscular dystrophy, a genetic muscle-wasting disease that rarely is seen in girls but affects one in 3,500 male babies, profoundly shortening life expectancy. It is the most common fatal disease that affects children. Muscle weakness begins in early childhood, often causing deformity of the arms, legs and spine. Heart and respiratory muscles often begin to fail before children reach early teen years. Although steroid medications which often are not well tolerated and other therapies may ease symptoms and delay the most severe effects, no disease-specific treatment exists, and patients rarely survive into their 30s. But in this case study of 10 boys with Duchenne muscular dystrophy, also called DMD, a single dose of a drug often prescribed for erectile dysfunction or pulmonary hypertension corrected defective blood vessel mechanisms and restored blood flow to exercising muscles. "The effects were immediate and dramatic, raising the question: If a single dose restores blood flow to muscle while the drug is in the patient's system, could … Continue reading
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