Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia
Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia
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Hoboken, USA: John Wiley & Sons, Inc
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English
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Publisher
Hoboken, USA: John Wiley & Sons, Inc
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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...
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Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia
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TN_cdi_doaj_primary_oai_doaj_org_article_31070a4fa3a24d09ab5ad48e0225c9e3
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_31070a4fa3a24d09ab5ad48e0225c9e3
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ISSN
2192-8312,2192-8304
E-ISSN
2192-8312
DOI
10.1002/jmd2.12198
