期刊文献+
任意字段
题名或关键词
题名
关键词
文摘
作者
第一作者
机构
刊名
分类号
参考文献
作者简介
基金资助
栏目信息

治疗苯丙酮尿症特异性药物二盐酸沙丙蝶呤 被引量：3

A specific drug to treat PKU: sapropterin dihydrochloride
原文传递
导出
摘要 二盐酸沙丙蝶呤是天然四氢生物蝶呤(BH4)经人工合成得到的二盐酸盐,能激活苯丙氨酸羟化酶(PAH),改善正常的苯丙氨酸(Phe)氧化代谢,降低患者的Phe水平,是首个治疗苯丙酮尿症的特异性药物。现综述其作用机制、药动学、临床研究及不良反应。 Sapropterin dihydrochloride （Kuvan） is a synthetic dihydrochloride salt from the naturally occurring BH4. Kuvan can activate PAH enzyme, improve the normal oxidative metabolism of Phe, and decrease Phe levels in patients. Kuvan is the first specific drug to treat PKU. This paper introduced the mechanism of action, pharmacokinetics, clinical study and adverse reactions of Kuvan.
作者 吴叶红 刘海净 王玉红
机构地区 开滦集团有限责任公司医院
出处 《中国新药杂志》 CAS CSCD 北大核心 2009年第6期478-479,共2页 Chinese Journal of New Drugs
关键词 二盐酸沙丙蝶呤 苯丙酮尿症 作用机制 临床研究 sapropterin dihydrochloride phenylketonuria mechanism of action clinical study
分类号 R589 [医药卫生—内分泌]
  • 相关文献

参考文献5

  • 1BURNETT JR. Sapropterin dihydrochloride (Kuvan/phenoptin) , an orally active synthetic form of BH4 for the treatment of phenylketonuria[ J]. IDrugs,2007,10( 11 ) :805 - 813.
  • 2MICHALS-MATALON K. Sapropterin dihydrochloride, 6-R-L-erythro-5,6,7,8-tetrahydrobiopterin, in the treatment of phenylketonuria [ J ]. Expert Opin Investig Drugs,2008,17 ( 2 ) : 245 - 251.
  • 3BURTON BK,GRANGE DK,MILANOWSKI A,et al. The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R-tetrahydrobiopterin) : a phase Ⅱ, muhicentre, open-label, screening study [ J ]. J Inherit Metab Dis,2007,30 ( 5 ) : 700 - 707.
  • 4DOGGRELL SA. Is sapropterin treatment suitable for all subjects with phenylketonuria? [ J ]. Expert Opin Pharmacother, 2008,9 (1):145 -147.
  • 5LEVY HL, MILANOWSKI A, CHAKRAPANI A, et al. Efficacy of sapropterln dlhydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase Ⅲ randomised placebo-controlled study [ J ]. Lancet, 2007,370 ( 9586 ) : 504 - 510.

同被引文献14

  • 1Sanford M, Keating GM. Sapropterin, a review of its use in the treatment of primary hyperphenylalaninaemia [J]. Drugs, 2009, 69 (4): 461-476.
  • 2Kimberlee MM. Sapropterin dihydroehloride, 6R-L-erythro- 5,6,7,8-tetrahydrobiopterin, in the treatment of phenylketo- nuria [J]. Expert Opin InvestigDrugs, 2008, 17 (2) : 245-251.
  • 3Hong H, Gage J, Chen C, et al. Method for the synthesi- zing sapropterin dihydrochloride: WO, 2013152609 [P]. 2013-10-17.
  • 4Moser R, Groehn V, Martin P, et al. Processes for preparing tetrahydrobiopterin, and analogs of tetrahydrobiopterin: WO, 2005049614 [P]. 2005-06-02.
  • 5Mori K, Kikuchi H. A new synthesis of (-) -biopterin employing 5-deoxy-L-ribose as a key intermediate [J]. Eur J Org Chem, 1989, (12) : 1267-1269.
  • 6Tazawa S. Method for producing L-biopterin- US, 20060142573A1 [P]. 2006-06-29.
  • 7Emanuele A, Mario M, Davide R, et al. Process for the preparation of pteridine derivatives: WO, 2011157388 [P]. 2011-12-22.
  • 8Katsuhide MO. Process for producing carbon-diminished aldose compound: WO, 2006070862 [P]. 2006-07-06.
  • 9Yokohama SH, Chiba TK. Preparation process of (6R) - tetrahydro-L-biopterin: US, 4713454 [P]. 1987-12- 1 5.
  • 10Randi KG. Unveiling the role of molecule-assisted homolysis: a mechanistic probe into the chemistry of a bicyclic peroxide [J]. J Org Chem, 2012, 77 (5): 2134-2141.

引证文献3

二级引证文献2

中国新药杂志
2009年 第6期

相关作者

内容加载中请稍等...

相关机构

内容加载中请稍等...

相关主题

内容加载中请稍等...

浏览历史

内容加载中请稍等...
;
使用帮助 返回顶部