Magazine article Science News

Steroid Slackens Pace of Muscular Dystrophy

Magazine article Science News

Steroid Slackens Pace of Muscular Dystrophy

Article excerpt

Steriod slackens pace of muscular dystrophy

A powerful steroid has poved its mettle as the first drug to slow progressive muscle weakening in youngsters with Duchenne's muscular dystrophy. Although the drug, called prednisone, carries serious side effects, researchers believe it may ultimately point the way to an equally effective but safer treatment.

"For the first time, we have a long-term study that shows [prednisone's] efficacy," says Gerald M. Fenichel of Vanderbilt University in Nashville, who presented the results this week at the American Academy of Neurology meeting in Boston. He cautions, however, that the drug cannot rid patients of the disease. "Prednisone is not a cure," he says.

One out of every 3,500 male infants in the United States has inherited a defective gene that causes Duchenne's muscular dystrophy. At about age 3, afflicted children develop a swaying or waddling motion when they walk. From that seemingly innocent early stage, the disease advances until weakened muscles can no longer hold the body upright. Even with physical theraphy, most victims need a wheelchair by age 12, and most die of heart or lung complications by age 20.

Several years ago, a study directed by Daniel B. Drachman of Johns Hopkins University in Baltimore hinted at prednisone's potential in boys with Duchenne's -- the most serious form of muscular dystrophy (SN: 8/22/87, p.120). Fenichel and his colleagues have now confirmed that early promise in a multicenter trial of 89 Duchenne's patients ranging from 7 to 14 years of age.

In a year-long experiment, Fenichel's team found that a daily doe of 0.75 milligrams of prednisone per kilogram of body weight seemed to offer the best short at fighting the muscle-sapping disorder. After that initial phase, they continued to treat the boys but had to lower the maximum dose for some patients because of harsh side effects. …

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