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A review of the application of asfotase alfa in the rare disease hypophosphatasia
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Qing-yang LIU1, Zi-jing HOU1, 2, Yu GE2, Shao-hong WANG1, Xin LIU1, Bo ZHANG1, Yan TANG1
Chinese Journal of New Drugs | 2023, 32(24) : 2526 - 2530
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Chinese Journal of New Drugs | 2023, 32(24): 2526-2530
A review of the application of asfotase alfa in the rare disease hypophosphatasia
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Qing-yang LIU1, Zi-jing HOU1, 2, Yu GE2, Shao-hong WANG1, Xin LIU1, Bo ZHANG1, Yan TANG1
Affiliations
  • 1Department of Pharmacy, Chinese Academy of Medical Sciences and Peking Union Medical College, Peking Union Medical Union Hospital, Beijing 100730, China
  • 2Department of Pharmacy, Dongzhimen Hospital, Beijing University of Chinese Medicine, Beijing 100700, China
Published: 2023-12-30
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Hypophosphatasia (HPP) is a rare hereditary endocrine system disease characterized by bone and/or tooth mineralization disorders accompanied by decreased serum alkaline phosphatase activity. Asfotase alfa (AA) received orphan drug approval by FDA as a recombinant bone-targeted human nonspecific alkaline phosphatase and was marketed in 2015 for the treatment of HPP. This paper reviews the mechanism of action, pharmacokinetic characteristics, efficacy, safety, usage and dosage of AA to provide reference and evidence support for the use of AA in the patients with HPP.

hypophosphatasia  /  rare disease  /  orphan drug  /  asfotase alfa
Qing-yang LIU, Zi-jing HOU, Yu GE, Shao-hong WANG, Xin LIU, Bo ZHANG, Yan TANG. A review of the application of asfotase alfa in the rare disease hypophosphatasia[J]. Chinese Journal of New Drugs, 2023 , 32 (24) : 2526 -2530 .
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  • Online Date：2026-03-05
  • Published：2023-12-30
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  • Accepted：2023-03-26
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Affiliations
    1Department of Pharmacy, Chinese Academy of Medical Sciences and Peking Union Medical College, Peking Union Medical Union Hospital, Beijing 100730, China
    2Department of Pharmacy, Dongzhimen Hospital, Beijing University of Chinese Medicine, Beijing 100700, China
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表12种不同金属材料的力学参数
科
Family		 属数
Number of
genus		 种数
Number of
species		 占总种数比例
Percentage of
total species (%)		 属
Genus		 种数
Number of
species		 占总种数比例
Percentage of total
species (%)
鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
小菇属 Mycena 11 5.26
光柄菇属 Pluteus 5 2.39
红菇属 Russula 17 8.13
栓菌属 Trametes 5 2.39
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