2.5-years of vamorolone treatment in Duchenne muscular dystrophy: Results of an open label long-term extension

被引：1

作者：

Hoffman, E. ^{[1
]}

Dang, U. ^{[1
]}

Clemens, P. ^{[2
]}

Gordish-Dressman, H. ^{[3
]}

Schwartz, B. ^{[4
]}

Mengle-Gaw, L. ^{[4
]}

Leinonen, M. ^{[5
]}

Smith, E. ^{[6
]}

Castro, D. ^{[7
]}

Kuntz, N. ^{[8
]}

Finkel, R. ^{[9
]}

Tulinius, M. ^{[10
]}

Nevo, Y. ^{[11
]}

Ryan, M. ^{[12
]}

Webster, R. ^{[13
]}

van den Anker, J. ^{[3
]}

Ward, L. ^{[14
]}

Damsker, J. ^{[15
]}

McDonald, C. ^{[16
]}

Guglieri, M. ^{[17
]}

Mah, J. ^{[18
]}

机构：

[1] SUNY Binghamton, Binghamton, NY USA

[2] Univ Pittsburgh, Pittsburgh, PA USA

[3] Childrens Natl Med Ctr, Washington, DC 20010 USA

[4] Camden Associates, St Louis, MO USA

[5] Santhera, Pratteln, Switzerland

[6] Duke Univ, Chapel Hill, NC USA

[7] UTSW, Dallas, TX USA

[8] Luries Childrens Hosp, Chicago, IL USA

[9] Nemours Childrens Hosp, Orlando, FL USA

[10] Queen Silvia Childrens Hosp, Gothenburg, Sweden

[11] Tel Aviv Univ, Tel Aviv, Israel

[12] Royal Childrens Hosp, Melbourne, Vic, Australia

[13] Childrens Hosp Westmead, Sydney, NSW, Australia

[14] Childrens Hosp Eastern Ontario, Ottawa, ON, Canada

[15] ReveraGen, Rockville, MD USA

[16] Univ Calif Davis, Davis, CA 95616 USA

[17] Univ Newcastle, Newcastle, England

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

来源：

NEUROMUSCULAR DISORDERS | 2021年 / 31卷

关键词：

D O I：

10.1016/j.nmd.2021.07.020

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

O.3a

引用

页码：S48 / S48

页数：1

共 50 条

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Hoffman, E.
Dang, U.
Clemens, P.
Gordish-Dressman, H.
Schwartz, B.
Mengle-Gaw, L.
Leinonen, M.
Smith, E.
Castro, D.
Kuntz, N.
Finkel, R.
Tulinius, M.
Nevo, Y.
Ryan, M.
Webster, R.
van den Anker, J.
Ward, L.
Damsker, J.
McDonald, C.
Guglieri, M.
Mah, J.
[J]. NEUROMUSCULAR DISORDERS, 2021, 31 : S93 - S94
[2] Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study
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[4] Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy A 30-Month Nonrandomized Controlled Open-Label Extension Trial
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