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α-GaIA酶替代疗法治疗Fabry病的临床应用与进展 被引量:4

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摘要 Fabry病是一种与X染色体连锁遗传的先天性α-半乳糖苷酶A(α-GaI A)缺乏疾病。重组人α-GaI A产品agalsidase-α和agalsidase-β的问世,给Fabry病患者的治疗带来历史性突破,使Fabry病患者正常存活变成现实。但目前α-GaI A酶替代疗法应用时间尚短,治疗病例数也不是很多,且价格昂贵。因此对长期α-GaI A酶替代治疗的疗效、安全性等还有待进一步研究。
出处 《世界临床药物》 CAS 2005年第2期75-78,共4页 World Clinical Drug
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  • 1L. Jardim,L. Vedolin,I. V. D. Schwartz,M. G. Burin,C. Cecchin,L. Kalakun,U. Matte,F. Aesse,C. Pitta-Pinheiro,J. Marconato,R. Giugliani. CNS involvement in Fabry disease: Clinical and imaging studies before and after 12 months of enzyme replacement therapy[J] 2004,Journal of Inherited Metabolic Disease(2):229~240

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