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Inborn errors of carbohydrate metabolism / Rare diseases / Glycogen storage disease type II / Lysosomal storage disease / Acid alpha-glucosidase / Enzyme replacement therapy / Glycogen / Medicine / Health / Hepatology


NIH Public Access Author Manuscript Neurotherapeutics. Author manuscript; available in PMC 2009 October 14. NIH-PA Author Manuscript
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Document Date: 2010-07-21 23:05:58

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City

Rotterdam / Paris / Munich / Framingham / /

Company

Genzyme Corporation / /

Continent

Europe / /

Country

Germany / France / United States / /

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Event

FDA Phase / /

Facility

Ludwig Maximilians University / Rockville Pike / Friedrich-Baur Institute / /

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IndustryTerm

active site / gene transfer systems / injection site / chemical conjugation / therapy of muscle disorders / expanded access protocol / characterized gene transfer systems / milk product / manufacturing / therapy for Pompe disease / chemical chaperone therapy / obvious site / satellite cell activation / /

MedicalCondition

advanced disease / hypertrophic cardiomyopathy / cardiac and respiratory failure / lysosome INTRODUCTION Pompe disease / lysosomal storage disorders / cardiac failure / Arthritis / coma / muscle weakness / autosomal recessive multi-system disorder / starvation / glycogen storage disease / muscle disorders / long-established disease / human diseases / infantile onset Pompe disease / late onset Pompe disease / Pompe disease / intractable fever / near-complete enzyme deficiency / progressive skeletal muscle weakness / immune-deficient GAA-KO/SCID / distal foot drop syndrome / cardiomyopathy / cancer / disease / respiratory failure / pulmonary function / rare disease / GAA-KO/SCID / less fatigue / lysosomal storage diseases / infection / viral pneumonia / deficiency / glycogen storage diseases / /

MedicalTreatment

EXPERIMENTAL THERAPIES / gene therapy / Enzyme replacement therapy / /

Organization

National Institute of Health / i.v. administration / Friedrich-Baur Institute / Department of Neurology / Ludwig Maximilians University Munich / European Medicines Agency / Intravenous administration / /

Person

Benedikt Schoser / Alberta Infant / Nina Raben / Victoria Hill / /

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Position

Author / Governor / /

Product

Myozyme / ERT / M6P / Europe / /

Technology

alpha / antibodies / gene therapy / expanded access protocol / /

URL

http /

SocialTag