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Glycogen storage disease type II / Lysosomal storage disease / Acid alpha-glucosidase / Newborn screening / Screening / Medical genetics / Medicine / Health / Rare diseases


EVIDENCE REVIEW: POMPE DISEASE Prepared for: THE ADVISORY COMMITTEE ON HERITABLE DISORDERS IN NEWBORNS AND CHILDREN FINAL V[removed]
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Document Date: 2014-12-04 01:12:52

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City

Medical College / Alixandra Knapp / Boston / Cambridge / Vienna / /

Company

Genzyme Corporation / MPH Evidence Review Group / Medline / Health Resources / /

Continent

North America / Europe / /

Country

Austria / Israel / /

Facility

Massachusetts General Hospital / Altarum Institute / Columbia University Medical College / Columbia University / National Taiwan University Hospital / Duke University / University Children's Hospital / National Library of Medicine Medical Subject Headings / University of Massachusetts / University of Michigan / /

IndustryTerm

organ systems / treatment of Pompe disease / treatment for infantile-onset Pompe disease / /

MedicalCondition

non-classic infantile-onset disease / glycogen storage disease type II / dysfunction / later-onset disease / muscle weakness / Cardiomegaly / glycogen storage disease / Complete deficiency / disorder / lysosomal storage disease / non-classic disease / illness / MS / infantile-onset Pompe disease / Pompe disease / cardiomyopathy / disease / late-onset disease / Left ventricular hypertrophy / untreated infantile Pompe disease / acid maltase deficiency / Cardiac disease / respiratory insufficiency / classic disease / /

MedicalTreatment

treatment protocol / enzyme replacement therapy / /

Organization

U.S. Department of Health and Human Services / Columbia University / University of Massachusetts / Durham NC / MGH Center for Child and Adolescent Health Policy / University of Michigan / Massachusetts General Hospital / Health Resources and Services Administration / National Taiwan University / Duke University / Durham / Child Health Bureau / Duke University / Altarum Institute / Services Administration / /

Person

James M. Perrin / Anne Comeau / Denise Queally / Alfred E. Slonim / Nancy Green / Priya Kishnani / Ellen Lipstein / Marsha Browning / Deborah Marsden / Lisa Prosser / Deeksha Bali / Olaf Bodamer / Paul Wuh-Liang / Alex R. Kemper / Joan Keutzer / Wuh-Liang Hwu / /

Position

investigator / Director / Global Medical Affairs –Myozyme / Vice-President / Chief / Medical Genetics / author / Professor / Director / Chairperson / Major / Representative / /

Product

thrive / Myozyme / /

ProvinceOrState

Maryland / New York / Michigan / North Carolina / Massachusetts / /

PublishedMedium

Natural History / Molecular Biology / /

Technology

alpha / /

SocialTag