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Pompe's disease: Enzyme replacement therapy

Publication ,  Journal Article
Koeberl, DD; Kishnani, PS
Published in: Drugs of the Future
December 1, 2007

The approval of Myozyme® (alglucosidase alfa; Genzyme) represented the first significant advance in therapy for Pompe's disease. Pompe's disease is the inherited deficiency of acid α-glucosidase (GAA), and hence the rationale for treatment with Myozyme®, a form of recombinant human (rh)GAA. Previously, the natural history of infantile Pompe's disease featured rapid progression of hypertrophic and dilated cardiomyopathy to death from cardiorespiratory failure by 1 year of age. The course of infantile Pompe's disease was significantly altered by enzyme replacement therapy (ERT) with rhGAA, which improved survival and ventilator-free survival, as well as reversing cardiomyopathy and improving motor development. Limitations of ERT include high dose requirements to treat the large skeletal muscle mass, suboptimal receptor-mediated uptake and complicating antibody responses to rhGAA therapy in a subset of patients. A randomized trial in late-onset Pompe's disease is under way, as an improvement in motor and pulmonary function has been reported in small trials. Copyright © 2007 Prous Science.

Duke Scholars

Published In

Drugs of the Future

DOI

ISSN

0377-8282

Publication Date

December 1, 2007

Volume

32

Issue

12

Start / End Page

1067 / 1070

Related Subject Headings

  • Medicinal & Biomolecular Chemistry
  • 3214 Pharmacology and pharmaceutical sciences
  • 1115 Pharmacology and Pharmaceutical Sciences
 

Citation

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Koeberl, D. D., & Kishnani, P. S. (2007). Pompe's disease: Enzyme replacement therapy. Drugs of the Future, 32(12), 1067–1070. https://doi.org/10.1358/dof.2007.032.12.1158295
Koeberl, D. D., and P. S. Kishnani. “Pompe's disease: Enzyme replacement therapy.” Drugs of the Future 32, no. 12 (December 1, 2007): 1067–70. https://doi.org/10.1358/dof.2007.032.12.1158295.
Koeberl DD, Kishnani PS. Pompe's disease: Enzyme replacement therapy. Drugs of the Future. 2007 Dec 1;32(12):1067–70.
Koeberl, D. D., and P. S. Kishnani. “Pompe's disease: Enzyme replacement therapy.” Drugs of the Future, vol. 32, no. 12, Dec. 2007, pp. 1067–70. Scopus, doi:10.1358/dof.2007.032.12.1158295.
Koeberl DD, Kishnani PS. Pompe's disease: Enzyme replacement therapy. Drugs of the Future. 2007 Dec 1;32(12):1067–1070.

Published In

Drugs of the Future

DOI

ISSN

0377-8282

Publication Date

December 1, 2007

Volume

32

Issue

12

Start / End Page

1067 / 1070

Related Subject Headings

  • Medicinal & Biomolecular Chemistry
  • 3214 Pharmacology and pharmaceutical sciences
  • 1115 Pharmacology and Pharmaceutical Sciences