Dystrophin restoration by exon 53 skipping in patients with Duchenne muscular dystrophy after viltolarsen treatment: phase 2 study update

被引：1

作者：

Clemens, P. ^{[1
]}

Rao, V. ^{[2
]}

Connolly, A. ^{[3
]}

Zaidman, C. ^{[3
]}

Harper, A. ^{[4
]}

Mah, J. ^{[5
]}

Morgenroth, L. ^{[6
]}

Yamashita, T. ^{[7
]}

Hoffman, E. ^{[8
]}

机构：

[1] Univ Pittsburgh, Pittsburgh, PA USA

[2] Lurie Childrens Hosp, Chicago, IL USA

[3] Washington Univ, St Louis, MO 63110 USA

[4] Virginia Commonwealth Univ, Richmond, VA 23284 USA

[5] Alberta Childrens Prov Gen Hosp, Calgary, AB, Canada

[6] TRiNDS, Kensington, PA USA

[7] NS Pharma, Paramus, NJ USA

[8] SUNY Binghamton, Binghamton, NY USA

来源：

关键词：

D O I：

10.1016/j.nmd.2019.06.451

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P.337

引用

页码：S165 / S166

页数：2