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Experimental Drug Offers Hope for Rare Bone Disease: Study
Date:3/7/2012

By Serena Gordon
HealthDay Reporter

WEDNESDAY, March 7 (HealthDay News) -- A new therapy may be the first to offer hope for children born with a rare disease that affects bone development, sometimes so severely that babies die because they're missing a rib cage to protect their lungs.

The inherited disorder is called hypophosphatasia, and the new medication is asfotase alfa. It works by replacing an enzyme that's missing in those with hypophosphatasia. Enzymes are substances responsible for speeding up certain chemical reactions. In hypophosphatasia, the missing enzyme is necessary for proper bone growth and normal metabolism.

A small study of babies and children younger than 3 who had debilitating or life-threatening hypophosphatasia found that treatment with asfotase alfa strengthened bones and improved lung function. After 48 weeks of treatment, many could start bearing weight on their legs and some infants were even taking their first steps.

"We saw striking improvements in these patients with severe hypophosphatasia who received the enzyme replacement," said the study's lead author, Dr. Michael Whyte, medical-scientific director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospitals for Children in St. Louis. Whyte is also a professor at Washington University School of Medicine in St. Louis, which conducted the study jointly with Shriners and other institutions.

Results of the research are published in the March 8 issue of the New England Journal of Medicine.

Severe hypophosphatasia affects about 1 in 100,000 babies born in the United States, according to the National Library of Medicine. It's estimated that more people may have the disease, but in far milder forms. The severity of the disease can range from life-threatening to simply causing dental problems in adults, according to background information in the article.

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