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ASGCT: Gene Therapy Shows Promise for Patients with Pompe Disease
Date:1/30/2010

New research in gene therapy is bringing doctors and patients closer to a comprehensive treatment for a rare disease portrayed in a new Hollywood film, according to the American Society of Gene & Cell Therapy.

Milwaukee (Vocus) January 30, 2010 -- New research in gene therapy is bringing doctors and patients closer to a comprehensive treatment for a rare disease portrayed in a new Hollywood film, according to the American Society of Gene & Cell Therapy.

“Extraordinary Measures” is based on the true story of John Crowley, a father struggling to find treatment for his children with Pompe disease, a rare, and often fatal, inherited disease that severely weakens the heart and lungs. Currently, the only approved treatment for Pompe is the enzyme-replacement medication Myozyme, which was ultimately developed by Genzyme Corporation.

In a study released this week in Molecular Therapy, ASGCT’s official journal, researchers at the University of Florida reported that gene therapy targeted at the diaphragms of mice with Pompe disease improved the ability of the mice to breathe independently.

This summer, six infants with the disease will receive the treatment. Although the treatment is not a cure, when used in conjunction with other therapies, like the drugs portrayed in the film, it is another step toward improving the lives of patients with Pompe disease.

“While the availability of Myozyme has been a life-saving therapy and a breakthrough for those living with Pompe disease, it must serve as a catalyst for continued innovation,” said Robert Mattaliano, PhD, group vice president of protein research and development at Genzyme. “Gene and cell-based therapies are at the forefront of such endeavors and bring the potential go to beyond replacing a missing enzyme by further improving the muscle repair process in this disease and oth
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