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Profile of asfotase alfa in the treatment of hypophosphatasia: design, development, and place in therapy
Hypophosphatasia (HPP) is a multi-systemic metabolic disorder caused by loss-of-function mutations in the ALPL gene that encodes the mineralization-associated enzyme, tissue-nonspecific alkaline phosphatase (TNSALP). HPP is characterized by defective bone and dental mineralization, leading to skelet...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Dove Medical Press
2018
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6161731/ https://www.ncbi.nlm.nih.gov/pubmed/30288020 http://dx.doi.org/10.2147/DDDT.S154922 |