Asfotase alfa therapy for children with hypophosphatasia

Background. Hypophosphatasia (HPP) is caused by loss-of-function mutation(s) of the gene that encodes the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP). Consequently, cell-surface deficiency of TNSALP phosphohydrolase activity leads to extracellular accumulation of inorganic pyrophos...

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Xehetasun bibliografikoak
Argitaratua izan da:JCI Insight
Egile Nagusiak: Whyte, Michael P., Madson, Katherine L., Phillips, Dawn, Reeves, Amy L., McAlister, William H., Yakimoski, Amy, Mack, Karen E., Hamilton, Kim, Kagan, Kori, Fujita, Kenji P., Thompson, David D., Moseley, Scott, Odrljin, Tatjana, Rockman-Greenberg, Cheryl
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society for Clinical Investigation 2016
Gaiak:
Clinical Medicine
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC5033855/
https://ncbi.nlm.nih.gov/pubmed/27699270
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.85971
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