Physical Function and Health‐Related Quality of Life in Adults Treated With Asfotase Alfa for Pediat...
Physical Function and Health‐Related Quality of Life in Adults Treated With Asfotase Alfa for Pediatric‐Onset Hypophosphatasia
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Hoboken, USA: John Wiley & Sons, Inc
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Language
English
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Hoboken, USA: John Wiley & Sons, Inc
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ABSTRACT
Hypophosphatasia (HPP) is a rare, inherited, metabolic disease characterized by tissue‐nonspecific alkaline phosphatase deficiency resulting in musculoskeletal and systemic clinical manifestations. This observational study evaluated the effectiveness of enzyme replacement therapy with asfotase alfa on physical function and health‐relate...
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Physical Function and Health‐Related Quality of Life in Adults Treated With Asfotase Alfa for Pediatric‐Onset Hypophosphatasia
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TN_cdi_doaj_primary_oai_doaj_org_article_ea23b38a0d6346e8b6b73e39ad7e4c06
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_ea23b38a0d6346e8b6b73e39ad7e4c06
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ISSN
2473-4039
E-ISSN
2473-4039
DOI
10.1002/jbm4.10395
