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Anti-Cancer Drug Fights Immune Reaction In Some Infants With Pompe Disease

Posted Oct 11 2012 10:32pm

Adding a third anti-cancer agent to a current drug cocktail appears to have contributed to dramatic improvement in three infants with the most severe form of Pompe disease — a rare, often-fatal genetic disorder characterized by low or no production of an enzyme crucial to survival.

Duke researchers previously pioneered the development of the first effective treatment for Pompe disease via enzyme replacement therapy (ERT). ERT relies on a manufactured enzyme/protein to act as a substitute for the enzyme known to be lacking in patients with a particular disease. In Pompe disease, ERT has been found to reduce heart and muscle damage caused by the absence of the enzyme.

In the new study, appearing online in the Oct. 11, 2012, edition of the journal Genetics in Medicine, the Duke team added a new step to the therapeutic regimen to address complications suffered by a subset of infants with Pompe disease who are treated with ERT.

 

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