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Lysosomal storage diseases / Orphan drugs / Glycogen storage disease type II / Hepatology / Alglucosidase alfa / Pharmaceutical Benefits Scheme / Amyotrophic lateral sclerosis / Clinical trial / Health / Medicine / Rare diseases


Document Date: 2014-10-01 21:56:22


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Company

Sanofi-Aventis Australia Pty Ltd / PBAC / Genzyme / /

Country

Germany / United States / Netherlands / Canada / Australia / United Kingdom / /

Currency

USD / /

Facility

Erasmus Medical Centre / /

IndustryTerm

rehabilitative services / therapy for patients with Pompe disease / treatment of late onset Pompe disease / /

MedicalCondition

inherited disorder / rapidly progressive disease / acid alfa-glucosidase deficiency / progressive muscle weakness / disease / Proposed Therapy Pompe disease / cardiac failure / single disease / late-onset Pompe disease / annual questionnaire assessing disease / steadily progressive disease / treatment of late onset Pompe disease / late onset Pompe disease / infantile-onset Pompe disease / Pompe disease / /

Organization

Australasian Society for Inborn Errors / Pharmaceutical Benefits Advisory Committee / Erasmus Medical Centre / Division of Sanofi-Aventis Australia Pty Ltd / Australian Government / Department of Veterans Affairs / Metabolism/ Australia New Zealand Association of Neurologists / International Pompe Association / /

Position

Governor / /

Product

Myozyme / /

URL

http /

SocialTag