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The DOI matches its Crossref record; 30 of the 30 references it cites were found in a registry or an index.

A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

Open access
van der Ploeg, Ans T., Clemens, Paula R., Corzo, Deyanira, Escolar, Diana M., Florence, Julaine, Groeneveld, Geert Jan, Herson, Serge, Kishnani, Priya S., Laforet, Pascal, Lake, Stephen L., Lange, Dale J., Leshner, Robert T., Mayhew, Jill E., Morgan, Claire, Nozaki, Kenkichi, Park, Dorothy J., Pestronk, Alan, Rosenbloom, Barry, Skrinar, Alison, van Capelle, Carine I., van der Beek, Nadine A., Wasserstein, Melissa, Zivkovic, Sasa A.·2010·New England Journal of Medicine
DOI 10.1056/nejmoa0909859
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Abstract

BACKGROUND: Pompe's disease is a metabolic myopathy caused by a deficiency of acid alpha glucosidase (GAA), an enzyme that degrades lysosomal glycogen. Late-onset Pompe's disease is characterized by progressive muscle weakness and loss of respiratory function, leading to early death. We conducted a randomized, placebo-controlled trial of alglucosidase alfa, a recombinant human GAA, for the treatment of late-onset Pompe's disease. METHODS: Ninety patients who were 8 years of age or older, ambulatory, and free of invasive ventilation were randomly assigned to receive biweekly intravenous alglucosidase alfa (20 mg per kilogram of body weight) or placebo for 78 weeks at eight centers in the United States and Europe. The two primary end points were distance walked during a 6-minute walk test and percentage of predicted forced vital capacity (FVC). RESULTS: At 78 weeks, the estimated mean changes from baseline in the primary end points favored alglucosidase alfa (an increase of 28.1+/-13.1 m on the 6-minute walk test and an absolute increase of 3.4+/-1.2 percentage points in FVC; P=0.03 and P=0.006, respectively). Similar proportions of patients in the two groups had adverse events, serious adverse events, and infusion-associated reactions; events that occurred only in patients who received the active study drug included anaphylactic reactions and infusion-associated reactions of urticaria, flushing, hyperhidrosis, chest discomfort, vomiting, and increased blood pressure (each of which occurred in 5 to 8% of the patients). CONCLUSIONS: In this study population, treatment with alglucosidase alfa was associated with improved walking distance and stabilization of pulmonary function over an 18-month period. (ClinicalTrials.gov number, NCT00158600.)

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Metadata

Authors
van der Ploeg, A. T., Clemens, P. R., Corzo, D., Escolar, D. M., Florence, J., Groeneveld, G. J., Herson, S., Kishnani, P. S., Laforet, P., Lake, S. L., Lange, D. J., Leshner, R. T., Mayhew, J. E., Morgan, C., Nozaki, K., Park, D. J., Pestronk, A., Rosenbloom, B., Skrinar, A., van Capelle, C. I., van der Beek, N. A., Wasserstein, M., & Zivkovic, S. A.
Venue
New England Journal of Medicine
Publisher
Massachusetts Medical Society
Published
2010-04-15
Volume
362
Issue
15
Pages
1396-1406
ISSN
  • 0028-4793 (ISSN-L)
  • 1533-4406
DOI
10.1056/nejmoa0909859
OpenAlex
W2159490726
Cited by
745
References
30
FWCI
39.00
Type
journal article
Language
en
Open access
bronze

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CoChat. (2026). Citation check: A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease. https://app.cochat.ai/papers/10.1056/nejmoa0909859
The paper
van der Ploeg, A. T., Clemens, P. R., Corzo, D., Escolar, D. M., Florence, J., Groeneveld, G. J., Herson, S., Kishnani, P. S., Laforet, P., Lake, S. L., Lange, D. J., Leshner, R. T., Mayhew, J. E., Morgan, C., Nozaki, K., Park, D. J., Pestronk, A., Rosenbloom, B., Skrinar, A., . . . Zivkovic, S. A. (2010). A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease. New England Journal of Medicine. https://doi.org/10.1056/nejmoa0909859

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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.

Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).

Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.