Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia

CONTEXT: Hypophosphatasia (HPP) is an inborn error of metabolism that, in its most severe perinatal and infantile forms, results in 50–100% mortality, typically from respiratory complications. OBJECTIVES: Our objective was to better understand the effect of treatment with asfotase alfa, a first-in-c...

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Pubblicato in:J Clin Endocrinol Metab
Autori principali: Whyte, Michael P., Rockman-Greenberg, Cheryl, Ozono, Keiichi, Riese, Richard, Moseley, Scott, Melian, Agustin, Thompson, David D., Bishop, Nicholas, Hofmann, Christine
Natura: Artigo
Lingua:Inglês
Pubblicazione: Endocrine Society 2016
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4701846/
https://ncbi.nlm.nih.gov/pubmed/26529632
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1210/jc.2015-3462
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