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：

Ryan, M. M. ^{[1
,2
,3
]}

De Vivo, D. C. ^{[4
,5
]}

Bertini, E. ^{[6
]}

Hwu, W. L. ^{[7
,8
]}

Crawford, T. O. ^{[9
]}

Swoboda, K. J. ^{[10
]}

Finkel, R. S. ^{[11
]}

Kirschner, J. ^{[12
]}

Kuntz, N. L. ^{[13
]}

Parsons, J. ^{[14
]}

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

Topaloglu, H. ^{[16
]}

Ben Omran, T. ^{[17
,18
,19
]}

Sansone, V. A. ^{[20
,21
]}

Jong, Y. J. ^{[22
]}

Shu, F. ^{[23
]}

Foster, R. ^{[24
]}

Bhan, I. ^{[25
]}

Fradette, S. ^{[26
]}

Farwell, W. ^{[26
]}

机构：

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

[2] Univ Melbourne, Melbourne, Vic, Australia

[3] Murdoch Childrens Res Inst, Dept Neurol, Melbourne, Vic, Australia

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

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

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

[7] Natl Taiwan Univ Hosp, Dept Med Genet, Taipei, Taiwan

[8] Natl Taiwan Univ Hosp, Dept Pediat, Taipei, Taiwan

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

[10] Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA

[11] Nemours Childrens Hosp, Dept Pediat, Orlando, FL USA

[12] Univ Hosp Bonn, Dept Neuropediat, Bonn, Germany

[13] Ann & Robert H Lurie Childrens Hosp Chicago, Div Neurol, Chicago, IL 60611 USA

[14] Univ Colorado, Sch Med, Dept Pediat, Aurora, CO USA

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

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

[17] Sidra Med Qatar Fdn, Dept Pediat, Doha, Qatar

[18] Hamad Med Corp, Div Clin & Metab Genet, Doha, Qatar

[19] Hamad Med Corp, Dept Pediat, Doha, Qatar

[20] Univ Milan, Neuromuscular Omniserv, Milan, Italy

[21] Univ Milan, Dept Biomed Sci Hlth, Milan, Italy

[22] Kaohsiung Med Univ, Grad Inst Clin Med, Kaohsiung, Taiwan

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

[24] Biogen, Biostat, Maidenhead, Berks, England

[25] Biogen, Drug Safety, Cambridge, MA USA

[26] Biogen, Clin Dev, Cambridge, MA USA

来源：

JOURNAL OF THE NEUROLOGICAL SCIENCES | 2019年 / 405卷

关键词：

D O I：

10.1016/j.jns.2019.10.1276

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

WCN19-1058

引用

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
Sansone, V. A.
De Vivo, D. C.
Bertini, E.
Hwu, W. -L.
Crawford, T. O.
Swoboda, K. J.
Finkel, R. S.
Kirschner, J.
Kuntz, N.
Parsons, J.
Ryan, M. M.
Butterfield, R. J.
Topaloglu, H.
Ben Omran, T.
Jong, Y. -J.
Shu, F.
Bevernage, S. P.
Johnson, K.
Foster, R.
Bhan, I.
Fradette, S.
Farwell, W.
[J]. EUROPEAN JOURNAL OF NEUROLOGY, 2019, 26 : 83 - 83
[3] Nusinersen in Infants who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy: Interim Results from the Phase 2 NURTURE Study
Butterfield, R.
De Vivo, D.
Bertini, E.
Hwu, W-L
Crawford, T.
Swoboda, K.
Finkel, R.
Kirschner, J.
Kuntz, N.
Parsons, J.
Ryan, M.
Topaloglu, H.
Ben Omran, T.
Sansone, V
Jong, Y-J
Shu, F.
Johnson, K.
Foster, R.
Bhan, I
Fradette, S.
Farwell, W.
[J]. ANNALS OF NEUROLOGY, 2019, 86 : S117 - S117
[4] Nusinersen in infants who initiate treatment in a presymptomatic stage of spinal muscular atrophy: interim results from the phase 2 NURTURE study
Ryan, M.
De Vivo, D.
Bertini, E.
Hwu, W.
Crawford, T.
Swoboda, K.
Finkel, R.
Kirschner, J.
Kuntz, N.
Parsons, J.
Butterfield, R.
Topaloglu, H.
Ben Omran, T.
Sansone, V.
Jong, Y.
Shu, F.
Foster, R.
Bhan, I.
Fradette, S.
Farwell, W.
[J]. NEUROMUSCULAR DISORDERS, 2019, 29 : S185 - S185
[5] Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study
Crawford, T.
De Vivo, D.
Bertini, E.
Hwu, W. L.
Foster, R.
Bhan, I
Farwell, W.
Reyna, S.
[J]. ANNALS OF NEUROLOGY, 2018, 84 : S392 - S392
[6] Nusinersen in Infants Who Initiatte Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study
De Vivo, Darryl C.
Topaloglu, Haluk
Swoboda, Kathryn J.
Bertini, Enrico
Hwu, Wuh-Liang
Crawford, Thomas O.
Foster, Richard
Bhan, Ishir
Fradette, Stephanie
Farwell, Wildon
Reyna, Sandra P.
[J]. NEUROLOGY, 2019, 92 (15)
[7] Nusinersen effect in infants who initiate treatment in a presymptomatic stage of SMA: NURTURE results
Crawford, T.
Ryan, M.
Kirschner, J.
Finkel, R.
Swoboda, K.
De Vivo, D.
Bertini, E.
Hwu, W.
Foster, R.
Ramirez-Schrempp, D.
Chin, R.
Farwell, W.
[J]. NEUROMUSCULAR DISORDERS, 2020, 30 : S125 - S125
[8] Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
De Vivo, Darryl C.
Bertini, Enrico
Swoboda, Kathryn J.
Hwu, Wuh-Liang
Crawford, Thomas O.
Finkel, Richard S.
Kirschner, Janbernd
Kuntz, Nancy L.
Parsons, Julie A.
Ryan, Monique M.
Butterfield, Russell J.
Topaloglu, Haluk
Ben-Omran, Tawfeg
Sansone, Valeria A.
Jong, Yuh-Jyh
Shu, Francy
Staropoli, John F.
Kerr, Douglas
Sandrock, Alfred W.
Stebbins, Christopher
Petrillo, Marco
Braley, Gabriel
Johnson, Kristina
Foster, Richard
Gheuens, Sarah
Bhan, Ishir
Reyna, Sandra P.
Fradette, Stephanie
Farwell, Wildon
[J]. NEUROMUSCULAR DISORDERS, 2019, 29 (11) : 842 - 856
[9] Interim Efficacy and Safety Results from the Phase 2 NURTURE Study Evaluating Nusinersen in Presymptomatic Infants with Spinal Muscular Atrophy
De Vivo, Darryl C.
Hwu, Wuh-Liang
Reyna, Sandra P.
Farwell, Wildon
Gheuens, Sarah
Sun, Peng
Zhong, Zhenshao John
Su, John
Schneider, Eugene
Bertini, Enrico
[J]. NEUROLOGY, 2017, 88
[10] One-year Outcomes Following Treatment with Nusinersen: Interim Results from the NURTURE Study of Presymptomatic Infants with Genetically Diagnosed Spinal Muscular Atrophy (SMA)
De Vivo, D.
Bertini, E.
Hwu, W-L
Foster, R.
Gheuens, S.
Farwell, W.
Reyna, S.
[J]. ANNALS OF NEUROLOGY, 2017, 82 : S265 - S266

← 1 2 3 4 5 →