Safety and efficacy of long term asfotase alfa treatment in childhood hypophosphatasia
Safety and efficacy of long term asfotase alfa treatment in childhood hypophosphatasia
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England: BMC
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Language
English
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England: BMC
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Hypophosphatasia (HPP) is a rare inherited disorder characterized by a deficiency of tissue-non-specific alkaline phosphatase (TNSALP) due to loss-of-function variants of the ALPL gene. HPP is characterized by an extremely variable age of onset and clinical presentation, largely depending on the type of genetic disruption. Childhood HPP commonly pr...
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Full title
Safety and efficacy of long term asfotase alfa treatment in childhood hypophosphatasia
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TN_cdi_doaj_primary_oai_doaj_org_article_a95b6f9c86de4bc399a634a09adbdc20
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_a95b6f9c86de4bc399a634a09adbdc20
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ISSN
1824-7288
E-ISSN
1824-7288
DOI
10.1186/s13052-025-01883-2
