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Updated: Jan 13, 2026

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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卢塞拉斯塔特,Fabry病的口服疗法:一个关键的随机第三期研究及其开放式扩展的结果

Peter Nordbeck1,2, Ozlem Goker-Alpan3, John A Bernat4

  • 1Department of Internal Medicine I, University Hospital Würzburg, Würzburg, Germany.

Nature communications
|January 10, 2026
PubMed
概括

卢塞拉斯塔特是法布里病 (FD) 的基质还原疗法,在第三期试验中没有缓解神经病痛. 虽然它降低了血Gb3水平,但它对疼痛和功能的影响需要进一步研究.

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