Long-term safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from the ASPIRE study and open-label extension

被引：0

作者：

Ramaswami, Uma ^{[1
]}

Font-Montgomery, Esperanza E. ^{[2
,3
]}

Goker-Alpan, Ozlem ^{[4
]}

Ortiz, Damara ^{[5
]}

Sanchez-Valle, Amarilis ^{[6
]}

Whitley, Chester B. ^{[7
]}

Wilcox, William R. ^{[8
]}

Jiang, Hai ^{[9
]}

Lawson, Lee Ann ^{[9
]}

Vosk, Jennie ^{[9
]}

Yang, Haichen ^{[9
]}

Hopkin, Robert J. ^{[10
]}

机构：

[1] Royal Free London NHS Fdn Trust, London, England

[2] Cent Michigan Univ, Mt Pleasant, MI USA

[3] Univ Pediatricians, Mt Pleasant, MI USA

[4] Lysosomal & Rare Disorders Res & Treatment Ctr, Fairfax, VA USA

[5] Univ Pittsburgh, Med Ctr, Pittsburgh, PA USA

[6] Univ S Florida, Tampa, FL USA

[7] Univ Minnesota, Minneapolis, MN USA

[8] Emory Univ, Atlanta, GA USA

[9] Amicus Therapeut Inc, Princeton, NJ USA

[10] Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA

来源：

关键词：

D O I：

10.1016/j.ymgme.2024.108910

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

283

引用

页数：2