Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pomp...
Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
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Publisher
England: BioMed Central Ltd
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Language
English
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Publisher
England: BioMed Central Ltd
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Scope and Contents
Contents
Pompe disease is a rare, progressive, metabolic disease, and the first treatable inheritable muscle disorder. Enzyme replacement therapy (ERT) with alglucosidase alfa is disease specific and the only medicinal product authorized for the treatment of Pompe disease. Costs of ERT are very high as for most orphan drugs. This study investigates the cost...
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Full title
Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
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TN_cdi_doaj_primary_oai_doaj_org_article_9cfbaaa5b920451e8ecd10d255dadfc6
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_9cfbaaa5b920451e8ecd10d255dadfc6
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-017-0731-0
