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Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

机译：药理学伴侣Migalastat在法布里疾病和Migalastat-Amencable Variants经典表型患者患者中的疗效：来自第3阶段随机，多中心，双盲临床试验和推广研究的数据

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Dominique P. Germain; Kathy Nicholls; Roberto Giugliani; Daniel G. Bichet; Derralynn A. Hughes; Laura M. Barisoni; Robert B. Colvin; J. Charles Jennette; Nina Skuban; Jeffrey P. Castelli; Elfrida Benjamin; Jay A. Barth; Christopher Viereck;
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1. Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study [J] . Germain Dominique P., Nicholls Kathy, Giugliani Roberto, Genetics in medicine . 2019,第9期

机译：药理伴侣Migalastat在Mabry疾病和Migalastat-Amerable Variancts经典表型患者患者中的疗效：来自第3阶段随机，多中心，双盲临床试验和扩展研究的数据
2. Efficacy and safety of migalastat, an oral pharmacologic chaperone for Fabry disease: results from two randomized phase 3 studies, FACETS and ATTRACT [J] . Feldt-Rasmussen Ulla, Giugliani Roberto, Germain Dominique P., Molecular genetics and metabolism . 2017,第1a2期

机译：migalastat的疗效和安全性，用于法布里疾病的口腔药理伴侣：两种随机相3研究，方面和吸引的结果
3. Efficacy and safety of migalastat, an oral pharmacologic chaperone for Fabry disease: results from two randomized phase 3 studies, FACETS and ATTRACT [J] . Feldt-Rasmussen Ulla, Giugliani Roberto, Germain Dominique P., Molecular genetics and metabolism . 2017,第1a2期

机译：migalastat的疗效和安全性，用于法布里疾病的口腔药理伴侣：两种随机相3研究，方面和吸引的结果
4. Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized multicenter double-blind clinical trial and extension study [O] . Dominique P. Germain, Kathy Nicholls, Roberto Giugliani, -1

机译：药物伴侣伴侣migalastat在具有法布里病经典表型和migalastat适宜变体的男性患者亚组中的功效：来自3期随机多中心双盲临床试验和扩展研究的数据
5. Correction: Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study [O] . 2018

机译：矫正：口服药理伴侣MIGALASTAT与酶替代疗法相比，在法布里疾病中：随机期III的18个月的结果吸引研究

Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

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