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Article

Safety and efficacy of treatment with asfotase alfa in patients with hypophosphatasia: Results from a Japanese clinical trial.

Clinical endocrinology - 1 Jul 2017

Kitaoka Taichi, Tajima Toshihiro, Nagasaki Keisuke, Kikuchi Toru, Yamamoto Katsusuke, Michigami Toshimi, Okada Satoshi, Fujiwara Ikuma, Kokaji Masayuki, Mochizuki Hiroshi, Ogata Tsutomu, Tatebayashi Koji, Watanabe Atsushi, Yatsuga Shuichi, Kubota Takuo, Ozono Keiichi

Abstract excerpt

OBJECTIVE: Hypophosphatasia (HPP) is a rare skeletal disease characterized by hypomineralization and low alkaline phosphatase activity. Asfotase alfa (AA) has been recently developed to treat HPP complications. This study evaluated its safety and efficacy in Japan. DESIGN: Open-label, multicentre, prospective trial. Patients were enrolled in 11 hospitals from June 2014 to July 2015. PATIENTS: Thirteen patients (9...

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Identifiers

PMID: 28374482
DOI: 10.1111/cen.13343

Genes

ALPL

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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