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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English
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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_proquest_miscellaneous_2526143569
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2526143569
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2192-8304
DOI
10.1002/jmd2.12198