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Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia

Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2526143569

Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia

About this item

Full title

Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia

Journal title

JIMD reports, 2021, Vol.59 (1), p.10-15

Language

English

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Scope and Contents

Contents

Hypophosphatasia (HPP) is a rare inherited metabolic disorder characterized by deficient activity of alkaline phosphatase, causing defective mineralization of bones and teeth. The symptoms vary from no symptoms to stillbirth or skeletal manifestations. Since 2015, asfotase alfa, an enzyme replacement treatment, has been approved for pediatric use i...

Alternative Titles

Full title

Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia

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Primary Identifiers

Record Identifier

TN_cdi_proquest_miscellaneous_2526143569

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2526143569

Other Identifiers

ISSN

2192-8304

DOI

10.1002/jmd2.12198

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