Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study

CONTEXT: Long-term data on enzyme replacement treatment of hypophosphatasia (HPP) are limited. OBJECTIVE: To evaluate efficacy and safety of asfotase alfa in patients aged ≤5 years with HPP followed for up to 6 years. DESIGN: Phase 2 open-label study (July 2010 to September 2016). SETTING: Twenty-tw...

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Bibliografski detalji
Izdano u:J Clin Endocrinol Metab
Glavni autori: Hofmann, Christine E, Harmatz, Paul, Vockley, Jerry, Högler, Wolfgang, Nakayama, Hideki, Bishop, Nick, Martos-Moreno, Gabriel Á, Moseley, Scott, Fujita, Kenji P, Liese, Johannes, Rockman-Greenberg, Cheryl
Format: Artigo
Jezik:Inglês
Izdano: Endocrine Society 2019
Teme:
Clinical Research Articles
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6530655/
https://ncbi.nlm.nih.gov/pubmed/30811537
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1210/jc.2018-02335
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