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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Detalhes bibliográficos
Publicado no:JCI Insight
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2016
Assuntos:
Clinical Medicine
Acesso em linha: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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