Treatment With A New Drug Strengthens The Muscles Of Patients With Pompe Disease
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About The Author

Dwight Koeberl is a PhD researcher at Duke University Medical Center | DUMC · Division of Medical Genetics, specializing in medical genetics and gene therapy.

 

                       

Treatment With A New Drug Strengthens The Muscles Of Patients With Pompe Disease

Pompe disease is a rare and potentially fatal metabolic myopathy (prevalence 1 in 20,000). In Pompe disease, the deficiency of lysosomal acid α-glucosidase (GAA) leads to accumulation of lysosomal glycogen and muscle damage. Enzyme replacement therapy (ERT) for Pompe disease (glycogen storage disease type II; acid maltase deficiency) with alglucosidase alfa (rhGAA; LumizymeR) was approved by the Food and Drug Administration in 2006. Although treatment with ERT can slow the progression of symptoms in Pompe disease patients, the limitations of...

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