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Acid alpha-glucosidase deficiency (Pompe disease)

Tokiko Fukuda1, Ashley Roberts, Paul H Plotz

  • 1Arthritis and Rheumatism Branch, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Clinical Center, Bethesda, MD 20892, USA.

Current Neurology and Neuroscience Reports
|January 16, 2007
PubMed
Summary

Enzyme replacement therapy using recombinant acid alpha-glucosidase (alglucosidase alfa) offers new hope for Pompe disease patients. This review examines the benefits and limitations of this groundbreaking treatment for glycogen storage disorder.

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