A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
About this item
Full title
Author / Creator
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 and Zivkovic, Sasa A
Publisher
Waltham, MA: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
Waltham, MA: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Pompe's disease is caused by a deficiency of acid alpha glucosidase, which degrades lysosomal glycogen. Late-onset Pompe's disease is characterized by progressive muscle weakness and loss of respiratory function, leading to early death. In this randomized, placebo-controlled trial of a recombinant human acid alpha glucosidase, walking distance impr...
Alternative Titles
Full title
A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
Authors, Artists and Contributors
Author / Creator
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
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_733887808
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_733887808
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa0909859
