A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
- Author
- VAN DER PLOEG, Ans T1 ; CLEMENS, Paula R2 ; LANGE, Dale J8 ; LESHNER, Robert T4 ; MAYHEW, Jill E4 ; MORGAN, Claire3 ; NOZAKI, Kenkichi5 ; PARK, Dorothy J9 ; PESTRONK, Alan5 ; ROSENBLOOM, Barry9 ; SKRINAR, Alison3 ; VAN CAPELLE, Carine I1 ; CORZO, Deyanira3 ; VAN DER BEEK, Nadine A1 ; WASSERSTEIN, Melissa8 ; ZIVKOVIC, Sasa A2 ; ESCOLAR, Diana M4 ; FLORENCE, Julaine5 ; JAN GROENEVELD, Geert3 ; HERSON, Serge6 ; KISHNANI, Priya S7 ; LAFORET, Pascal6 ; LAKE, Stephen L3
[1] Departments of Pediatrics, Internal Medicine, Clinical Genetics, Neurology, and Hospital Pharmacy, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands
[2] Department of Neurology, University of Pittsburgh, and Neurology Service, Department of Veterans Affairs Medical Center, Pittsburgh, United States
[3] Genzyme, Cambridge, MA, United States
[4] Department of Neurology-Center for Genetic Medicine, Children's National Medical Center, Washington, DC, United States
[5] Department of Neurology, Washington University School of Medicine, St. Louis, United States
[6] Centre de Référence Pathologie Neuromusculaire Paris-Est, Hôpital Pitié-Salpêtrière, Assistance Publique-Hôpitaux de Paris, Paris, France
[7] Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, NC, United States
[8] Department of Genetics and Genomic Sciences, Mount Sinai School of Medicine, New York, United States
[9] Tower Hematology Oncology, Beverly Hills, CA, United States - Source
The New England journal of medicine. 2010, Vol 362, Num 15, pp 1396-1406, 11 p ; ref : 34 ref
- CODEN
- NEJMAG
- ISSN
- 0028-4793
- Scientific domain
- General medicine general surgery
- Publisher
- Massachusetts Medical Society, Waltham, MA
- Publication country
- United States
- Document type
- Article
- Language
- English
- Keyword (fr)
- Age apparition Glucide Glycogénose de Pompe Médecine Randomisation Tardif Enzymopathie Maladie héréditaire Maladie métabolique
- Keyword (en)
- Age of onset Carbohydrate Glycogenosis II Pompe Medicine Randomization Late Enzymopathy Genetic disease Metabolic diseases
- Keyword (es)
- Edad aparición Glúcido Glicogenosis II Pompe Medicina Aleatorización Tardío Enzimopatía Enfermedad hereditaria Metabolismo patología
- Classification
- Pascal
- 002 Biological and medical sciences / 002B Medical sciences / 002B22 Metabolic diseases / 002B22D Errors of metabolism / 002B22D03 Carbohydrates (enzymatic deficiencies). Glycogenosis
- Discipline
- Generalities in medical sciences Metabolic diseases
- Origin
- Inist-CNRS
- Database
- PASCAL
- INIST identifier
- 22637514
Sauf mention contraire ci-dessus, le contenu de cette notice bibliographique peut être utilisé dans le cadre d’une licence CC BY 4.0 Inist-CNRS / Unless otherwise stated above, the content of this bibliographic record may be used under a CC BY 4.0 licence by Inist-CNRS / A menos que se haya señalado antes, el contenido de este registro bibliográfico puede ser utilizado al amparo de una licencia CC BY 4.0 Inist-CNRS
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DOI
10.1056/NEJMoa0909859 
https://dx.doi.org/10.1056/NEJMoa0909859
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