Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update

被引：0

作者：

Day, John W. ^{[1
]}

Chiriboga, Claudia A. ^{[2
]}

Crawford, Thomas O. ^{[3
]}

Darras, Basil T. ^{[4
]}

Finkel, Richard S. ^{[5
]}

Connolly, Anne M. ^{[6
,7
,8
,9
]}

Iannaccone, Susan T. ^{[10
]}

Kuntz, Nancy L. ^{[11
]}

Pena, Loren D. M. ^{[12
]}

Shieh, Perry B. ^{[13
]}

Smith, Edward C. ^{[14
]}

Schultz, Meredith ^{[15
]}

Feltner, Douglas E. ^{[15
]}

Tauscher-Wisniewski, Sitra ^{[15
]}

Ogrinc, Francis G. ^{[15
]}

Shah, Ankita ^{[15
]}

Ouyang, Haojun ^{[15
]}

Macek, Thomas ^{[15
]}

Kernbauer, Elaine ^{[15
]}

Sproule, Douglas M. ^{[15
]}

Mendell, Jerry R. ^{[6
,7
]}

机构：

[1] Stanford Univ, Med Ctr, Dept Neurol, Stanford, CA 94305 USA

[2] Columbia Univ, Med Ctr, Div Pediat Neurol, New York, NY 10027 USA

[3] Johns Hopkins Med, Dept Neurol, Baltimore, MD USA

[4] Boston Childrens Hosp, Dept Neurol, Boston, MA USA

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

[6] Ohio Univ, Dept Pediat, Athens, OH 45701 USA

[7] Ohio Univ, Dept Neurol, Athens, OH 45701 USA

[8] Nationwide Childrens Hosp, Ctr Gene Therapy, Orlando, FL USA

[9] Nationwide Childrens Hosp, Dept Neurol, Orlando, FL USA

[10] Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, Dallas, TX USA

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

[12] Cincinnati Childrens Hosp, Div Human Genet, Cincinnati, OH USA

[13] Univ Calif Los Angeles, David Geffen Sch Med, Dept Neurol, Los Angeles, CA 90024 USA

[14] Duke Univ, Sch Med, Dept Pediat, Durham, NC 27706 USA

[15] AveXis Inc, Bannockburn, IL USA

来源：

NEUROLOGY | 2020年 / 94卷 / 15期

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：

1828

引用

页数：5

共 50 条

[1] Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy Type 1 (SMA1): Pivotal phase 3 study (STR1VE) update
Day, J.
Chiriboga, C.
Crawford, T.
Darras, B.
Finkel, R.
Connolly, A.
Iannaccone, S.
Kuntz, N.
Pena, L.
Schultz, M.
Shieh, P.
Smith, E.
Feltner, D.
Ogrinc, F.
Ouyang, H.
Macek, T.
Kernbauer, E.
Sproule, D.
Mendell, J.
NEUROMUSCULAR DISORDERS, 2019, 29 : S183 - S183
[2] Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update
Day, J.
Chiriboga, C.
Crawford, T.
Darras, B.
Finkel, R.
Connolly, A.
Iannaccone, S.
Kuntz, N.
Pena, L.
Schultz, M.
Shieh, P.
Smith, E.
Feltner, D.
Ogrinc, F.
Shah, A.
Ouyang, H.
Macek, T.
Kernbauer, E.
L'Italien, J.
Sproule, D.
Kaspar, B.
Mendell, J.
ANNALS OF NEUROLOGY, 2019, 86 : S118 - S121
[3] Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1: Pulmonary and Ventilatory Findings from the Pivotal Phase 3 US Study (STR1VE)
Shell, R.
Day, J. W.
Chiriboga, C. A.
Crawford, T. O.
Darras, B. T.
Finkel, R. S.
Connolly, A. M.
Iannaconne, S. T.
Kuntz, N. L.
Pena, L. D. M.
Shieh, P. B.
Smith, E. C.
Kwon, J. M.
Zaidman, C.
Schultz, M.
Feltner, D. E.
Tauscher-Wisniewski, S.
Ogrinc, F. G.
Thomasma, P.
Ouyang, H.
Macek, T. A.
Kernbauer, E.
Sproule, D. M.
Mendell, J. R.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2020, 201
[4] ONASEMNOGENE ABEPARVOVEC GENE-REPLACEMENT THERAPY (GRT) FOR SPINAL MUSCULAR ATROPHY TYPE 1 (SMA1): PRELIMINARY PULMONARY AND VENTILATORY FINDINGS FROM THE PHASE 3 STUDY (STR1VE)
Shell, R.
Day, J. W.
Chiriboga, C. A.
Crawford, T. O.
Darras, B. T.
Finkel, R. S.
Connolly, A. M.
Iannaccone, S. T.
Kuntz, N. L.
Pena, L. D. M.
Shieh, P. B.
Smith, E. C.
Kausar, I.
Schultz, M.
Feltner, D. E.
Ogrinc, F. G.
Macek, T. A.
Kernbauer, E.
L'Italien, J.
Sproule, D. M.
Kaspar, B. K.
Mendell, J. R.
THORAX, 2019, 74 : A42 - A42
[5] ONASEMNOGENE ABEPARVOVEC-XIOI GENE-REPLACEMENT THERAPY IN PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY (SMA): SPR1NT STUDY UPDATE
Strauss, Kevin A.
Swoboda, Kathryn J.
Farrar, Michelle A.
McMillan, Hugh J.
Parsons, Julie
Krueger, Jena M.
Iannaccone, Susan T.
Chiriboga, Claudia A.
Kwon, Jennifer M.
Saito, Kayoko
Scoto, Mariacristina
Schultz, Meredith
Kernbauer, Elaine
Farrow, Marcia
Ogrinc, Francis G.
Kavanagh, Sarah
Feltner, Douglas E.
McGill, Bryan E.
Meriggioli, Matthew
L'ltalien, James
Sproule, Douglas M.
Muntoni, Francesco
MUSCLE & NERVE, 2019, 60 : S13 - S13
[6] Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy in Presymptomatic Spinal Muscular Atrophy: SPR1NT Study Update
Strauss, Kevin A.
Farrar, Michelle A.
Swoboda, Kathryn J.
Saito, Kayoko
Chiriboga, Claudia A.
Finkel, Richard S.
Iannaccone, Susan T.
Krueger, Jena M.
Kwon, Jennifer M.
McMillan, Hugh J.
Servais, Laurent
Mendell, Jerry R.
Parsons, Julie
Scoto, Mariacristina
Shieh, Perry B.
Zaidman, Craig
Schultz, Meredith
Kernbauer, Elaine
Joshi, Shivani
Ogrinc, Francis G.
Kavanagh, Sarah
Feltner, Douglas E.
Tauscher-Wisniewski, Sitra
McGill, Bryan E.
Sproule, Douglas M.
Muntoni, Francesco
NEUROLOGY, 2020, 94 (15)
[7] Avxs-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update
Day, John W.
Chiriboga, Claudia A.
Crawford, Thomas O.
Darras, Basil T.
Finkel, Richard S.
Connolly, Anne M.
Iannaccone, Susan T.
Kuntz, Nancy L.
Pena, Loren D. M.
Schultz, Meredith
Shieh, Perry B.
Smith, Edward C.
Farrar, Michelle
Feltner, Douglas E.
Ogrinc, Francis G.
Macek, Thomas A.
Kernbauer, Elaine
Muehring, Lynlee M.
L'Italien, James
Sproule, Douglas M.
Kaspar, Brian K.
Mendell, Jerry R.
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 2019, 90 (E7):
[8] AVXS-101 Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update
Day, John W.
Chiriboga, Claudia A.
Crawford, Thomas O.
Darras, Basil T.
Finkel, Richard S.
Connolly, Anne M.
Iannaccone, Susan T.
Kuntz, Nancy L.
Pena, Loren D. M.
Schultz, Meredith
Shieh, Perry B.
Smith, Edward C.
Feltner, Douglas E.
Ogrinc, Francis G.
Macek, Thomas A.
Wells, Courtney
Muehring, Lynlee M.
L'Italien, James
Sproule, Douglas M.
Kaspar, Brian K.
Mendell, Jerry R.
NEUROLOGY, 2019, 92 (15)
[9] Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): Global pivotal phase 3 study program (STR1VE-US, STR1VE-EU, STR1VE-AP)
Mercuri, E.
Baranello, G.
Day, J. W.
Bruno, C.
Corti, S.
Chiriboga, C. A.
Crawford, T. O.
Darras, B. T.
Finkel, R. S.
Connolly, A. M.
Iannaccone, S. T.
Kuntz, N. L.
Masson, R.
Pena, L. D. M.
Baldinetti, F.
Schultz, M.
Shieh, P. B.
Smith, E. C.
Saito, K.
Scoto, M.
Spector, S. A.
Truncated, A. Authors
Sproule, D. M.
Mendell, J. R.
Muntoni, F.
JOURNAL OF THE NEUROLOGICAL SCIENCES, 2019, 405
[10] Onasemnogene Abeparvovec-xioi Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update
Strauss, K. A.
Farrar, M. A.
Swoboda, K. J.
Saito, K.
Chiriboga, C. A.
Finkel, R. S.
Iannaccone, S. T.
Krueger, J. M.
Kwon, J. M.
McMillan, H. J.
Servais, L.
Mendell, J. R.
Parsons, J.
Scoto, M.
Shieh, P. B.
Zaidman, C.
Schultz, M.
Baldinetti, F.
Ogrinc, F. G.
Kavanagh, S.
Feltner, D. E.
Tauscher-Wisniewski, S.
McGill, B. E.
Sproule, D. M.
Muntoni, F.
EUROPEAN JOURNAL OF NEUROLOGY, 2020, 27 : 165 - 166

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