Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial

被引：3

作者：

Clemens, Paula R. ^{[1
,2
]}

Mozaffar, Tahseen ^{[3
]}

Schoser, Benedikt ^{[4
]}

Bratkovic, Drago ^{[5
]}

Byrne, Barry J. ^{[6
]}

Goker-Alpan, Ozlem ^{[7
]}

Roberts, Mark ^{[8
]}

Schwenkreis, Peter. ^{[9
]}

Sivakumar, Kumaraswamy ^{[10
]}

van der Ploeg, Ans T. ^{[11
]}

Wright, Jacquelyn ^{[12
]}

Sitaraman, Sheela ^{[12
]}

Barth, Jay A. ^{[12
]}

Lagast, Hjalmar ^{[12
]}

Kishnani, Priya ^{[13
]}

Ming, Xue ^{[14
]}

机构：

[1] Univ Pittsburgh, Pittsburgh, PA USA

[2] Dept Vet Affairs Med Ctr, Pittsburgh, PA USA

[3] Univ Calif Irvine, Irvine, CA USA

[4] Ludwig Maximilians Univ Munchen, Friedrich Baur Inst, Neurol Klin, Munich, Germany

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

[6] Univ Florida, Gainesville, FL USA

[7] O&O Alpan LLC, Fairfax, VA USA

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

[9] Univ Klinikum Bergmannsheil, Neurol Klin & Poliklin Berufsgenossenschaftlichen, Bochum, Germany

[10] Neuromuscular Res Ctr, Phoenix, AZ USA

[11] Erasmus MC, Rotterdam, Netherlands

[12] Amicus Therapeut Inc, Cranbury, NJ USA

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

[14] Rutgers New Jersey Med Sch, Newark, NJ USA

来源：

关键词：

D O I：

10.1016/j.ymgme.2018.12.084

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：S40 / S41

页数：2