Hypophosphatasia - Pathophysiological understanding, preclinical data looking beyond the skeleton, and upcoming treatments
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Hypophosphatasia (HPP) is the genetic disorder caused by loss-of-function mutations in the ALPL gene that encodes tissue-nonspecific alkaline phosphatase (TNAP), an enzyme essential for physiological skeletal/dental mineralization. In HPP, TNAP deficiency lead... ...
