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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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| Izdano u: | J Clin Endocrinol Metab |
|---|---|
| Glavni autori: | , , , , , , , , , , |
| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
Endocrine Society
2019
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| Teme: | |
| 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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