Avalglucosidase alfa improves motor and respiratory function in late-onset Pompe disease: A COMET win-ratio analysis

被引：0

作者：

Diaz-Manera, Jordi ^{[1
]}

Dimachkie, Mazen M. ^{[2
]}

Ishak, Jack ^{[3
]}

Caro, Jaime ^{[4
]}

Hamed, Alaa ^{[5
]}

Franca, Lionel Riou ^{[6
]}

Thibault, Nathan ^{[5
]}

Shukla, Pragya ^{[3
]}

Berger, Kenneth I. ^{[7
]}

Boentert, Matthias ^{[8
]}

机构：

[1] Newcastle Univ, John Walton Muscular Dystrophy Res Ctr, Ctr Life, Newcastle Upon Tyne, England

[2] Univ Kansas, Dept Neurol, Med Ctr, Kansas City, KS USA

[3] Evidera, Montreal, PQ, Canada

[4] Evidera, Waltham, MA USA

[5] Sanofi, Cambridge, MA USA

[6] Sanofi, Paris, France

[7] New York Univ, Div Pulm Crit Care & Sleep Med, Grossman Sch Med, New York, NY USA

[8] Munster Univ Hosp, Dept Neurol, Munster, Germany

来源：

MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2022.107076

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：34 / 34

页数：1

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