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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Bibliografiska uppgifter
I publikationen:JCI Insight
Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society for Clinical Investigation 2016
Ämnen:
Clinical Medicine
Länkar: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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