Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory patients with Pompe disease: An open-label phase I/ II study (ATB200-02)

被引：2

作者：

Byrne, Barry J. ^{[1
]}

Schoser, Benedikt ^{[2
]}

Kishnani, Priya ^{[3
]}

Bratkovic, Drago ^{[4
]}

Clemens, Paula R. ^{[5
]}

Goker-Alpan, Ozlem ^{[6
]}

Ming, Xue ^{[7
]}

Roberts, Mark ^{[8
]}

van der Ploeg, Ans T. ^{[9
]}

Goldman, Mitchell ^{[10
]}

Wright, Jacquelyn ^{[10
]}

Holdbrook, Fred ^{[10
]}

Jain, Vipul ^{[10
]}

Sitaraman, Sheela ^{[10
]}

Wasfi, Yasmine ^{[10
]}

Mozaffar, Tahseen ^{[11
]}

机构：

[1] Univ Florida, Gainesville, FL USA

[2] Ludwig Maximilians Univ Munchen, Friedrich Baur Inst, Neurolog Klin, Munich, Germany

[3] Duke Univ, Med Ctr, Durham, NC USA

[4] Royal Adelaide Hosp, PARC Res Clin, Adelaide, SA, Australia

[5] Univ Pittsburgh, Sch Med, Dept Neurol, Neurol,Med Serv Line,VA Pittsburgh Healthcare Sys, Pittsburgh, PA USA

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

[7] Rutgers New Jersey Med Sch, Dept Neurol, Newark, NJ USA

[8] Salford Royal NHS Fdn Trust, Salford, England

[9] Erasmus MC Univ Med Ctr, Rotterdam, Netherlands

[10] Amicus Therapeut Inc, Philadelphia, PA USA

[11] Univ Calif Irvine, Irvine, CA USA

来源：

关键词：

D O I：

10.1016/j.ymgme.2022.107042

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：21 / 22

页数：2