Nusinersen in infants who initiate treatment in a presymptomatic stage of Spinal Muscular Atrophy (SMA): interim results from the phase 2 NURTURE study

被引：0

作者：

Sansone, V. A. ^{[1
,2
]}

De Vivo, D. C. ^{[3
,4
]}

Bertini, E. ^{[5
]}

Hwu, W. -L. ^{[6
]}

Crawford, T. O. ^{[7
]}

Swoboda, K. J. ^{[8
]}

Finkel, R. S. ^{[9
]}

Kirschner, J. ^{[10
]}

Kuntz, N. ^{[11
]}

Parsons, J. ^{[12
]}

Ryan, M. M. ^{[13
,14
]}

Butterfield, R. J. ^{[15
]}

Topaloglu, H. ^{[16
]}

Ben Omran, T. ^{[17
,18
]}

Jong, Y. -J. ^{[19
,20
,21
]}

Shu, F. ^{[22
]}

Bevernage, S. P. ^{[23
]}

Johnson, K. ^{[23
]}

Foster, R. ^{[24
]}

Bhan, I. ^{[23
]}

Fradette, S. ^{[23
]}

Farwell, W. ^{[23
]}

机构：

[1] Univ Milan, Dept Biomed Sci Hlth, Neuromuscular Omniserv, Milan, Italy

[2] Univ Milan, Neurorehabil Unit, Milan, Italy

[3] Columbia Univ, Irving Med Ctr, Dept Neurol, New York, NY USA

[4] Columbia Univ, Dept Pediat, Irving Med Ctr, New York, NY 10027 USA

[5] Postgrad Bambino Gesu Childrens Res Hosp, Unit Neuromuscular & Neurodegenerat Disorders, Rome, Italy

[6] Natl Taiwan Univ Hosp, Dept Med Genet & Pediat, Taipei, Taiwan

[7] Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA

[8] Massachusetts Gen Hosp, Dept Neurol, Ctr Genom Med, Boston, MA 02114 USA

[9] Nemours Childrens Hosp, Dept Pediat, Div Neurol, Orlando, FL USA

[10] Univ Freiburg, Med Ctr, Freiburg, Germany

[11] Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA

[12] Univ Colorado, Childrens Hosp Colorado, Aurora, CO USA

[13] Univ Melbourne, Royal Childrens Hosp, Melbourne, Vic, Australia

[14] Murdoch Childrens Res Inst, Melbourne, Vic, Australia

[15] Univ Utah, Dept Pediat & Neurol, Salt Lake City, UT USA

[16] Hacettepe Univ, Dept Pediat Neurol, Ankara, Turkey

[17] Weill Cornell Med Coll Qatar, Dept Pediat, Sect Clin & Metab Genet, Doha, Qatar

[18] Hamad Med Corp, Doha, Qatar

[19] Kaohsiung Med Univ, Coll Med, Grad Inst Clin Med, Dept Pediat, Kaohsiung, Taiwan

[20] Kaohsiung Med Univ, Coll Med, Grad Inst Clin Med, Dept Lab Med, Kaohsiung, Taiwan

[21] Kaohsiung Med Univ, Kaohsiung Med Univ Hosp, Kaohsiung, Taiwan

[22] Univ Calif Los Angeles, David Geffen Sch Med, Dept Neurol, Los Angeles, CA 90095 USA

[23] Biogen, Cambridge, MA USA

[24] Biogen, Maidenhead, Berks, England

来源：

EUROPEAN JOURNAL OF NEUROLOGY | 2019年 / 26卷

关键词：

D O I：

暂无

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

O3110

引用

页码：83 / 83

页数：1

共 50 条

[1] Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Results From the Phase 2 NURTURE Study
Finkel, Richard S.
De Vivo, Darryl C.
Swoboda, Kathryn J.
Bertini, Enrico
Hwu, Wuh-Liang
Foster, Richard
Bhan, Ishir
Fradette, Stephanie
Farwell, Wildon
[J]. NEUROLOGY, 2020, 94 (15)
[2] Nusinersen in infants who initiate treatment in a presymptomatic stage of spinal muscular atrophy (SMA): Interim results from the phase 2 nurture study
Ryan, M. M.
De Vivo, D. C.
Bertini, E.
Hwu, W. L.
Crawford, T. O.
Swoboda, K. J.
Finkel, R. S.
Kirschner, J.
Kuntz, N. L.
Parsons, J.
Butterfield, R. J.
Topaloglu, H.
Ben Omran, T.
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Jong, Y. J.
Shu, F.
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