伊米苷酶（Cerezyme），用于治疗戈谢病—静脉注射；β-半乳糖苷酶（Fabrazyme），用于治疗法布里病—静脉注射；酸性α-葡萄糖苷酶（Myozyme），用于治疗蓬佩病—静脉注射；拉罗尼酶（Aldurazyme），用于治疗粘...
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国外临床研究结果显示，给予病人注射外源性的酶（Fabrazyme）后，可明显改善病人疼痛等症状，延缓肾脏和心脏损害的进展，减少鞘糖脂类物质在组织中的沉积。
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Now Genzyme plans to replicate this strategy with Fabrazyme and Aldurazyme, two drugs which target ultra-rare genetic diseases.
FORBES: Magazine Article
Sinai School of Medicine, which was awarded an NIH grant and invented Fabrazyme (see here and here).
FORBES: Shire Ends Fabry Drug Bid, Giving Sanofi A Boost
Of course, Shire may also calculate there is less upside now that Fabrazyme production has resumed and passing the FDA gauntlet could become expensive.
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