Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global...
Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry
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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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Contents
Hypophosphatasia (HPP) is a rare inherited disease caused by deficient activity of tissue-nonspecific alkaline phosphatase. Many adults with HPP have a high burden of disease, experiencing chronic pain, fatigue, limited mobility, and dental issues, contributing to decreased health-related quality of life (HRQoL). HPP may be treated with the enzyme...
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Full title
Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry
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TN_cdi_doaj_primary_oai_doaj_org_article_37968c05241044a7ae66037fae804ebb
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_37968c05241044a7ae66037fae804ebb
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-024-03048-6
