Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial

被引：0
|
作者：
Mendell, J. [1 ]
Shell, R. [1 ]
Lehman, K. [1 ]
McColly, M. [1 ]
Lowes, L. [1 ]
Alfano, L. [1 ]
Miller, N. [1 ]
Iammarino, M. [1 ]
Church, K. [1 ]
Ogrinc, F. [2 ]
Ouyang, H. [2 ]
Kernbauer, E. [2 ]
Shah, S. [2 ]
L'Italien, J. [2 ]
Sproule, D. [2 ]
Feltner, D. [2 ]
Al-Zaidy, S. [1 ]
机构：
[1] Nationwide Childrens Hosp, Columbus, OH USA
[2] AveXis Inc, Bannockburn, IL USA
来源：
NEUROMUSCULAR DISORDERS | 2019年 / 29卷
关键词：
D O I：
10.1016/j.nmd.2019.06.513
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
P.351
引用
收藏
页码：S184 / S184
页数：1
相关论文
共 50 条
  • [41] ONASEMNOGENE ABEPARVOVEC GENE THERAPY FOR SPINAL MUSCULAR ATROPHY TYPE 1: PHASE 3 STUDY (STR1VE-US)
    Day, J. W.
    Finkel, R. S.
    Connolly, A. M.
    Darras, B. T.
    Iannaccone, S. T.
    Kuntz, N. L.
    Pena, L. D. M.
    Smith, E. C.
    Chiriboga, C. A.
    Crawford, T. O.
    Shieh, P. B.
    Kwon, J. M.
    Zaidman, C. M.
    Schultz, M.
    Kausar, I.
    Chand, D.
    Tauscher-Wisniewski, S.
    Ouyang, H.
    Macek, T. A.
    Mendell, J. R.
    THORAX, 2021, 76 : A10 - A11
  • [42] Adeno-Associated Virus Serotype 9 (AAV9) Antibodies in Patients With Spinal Muscular Atrophy (SMA) Screened for Treatment With Gene-Replacement Therapy (GRT) Onasemnogene Abeparvovec
    Day, J.
    Finkel, R.
    Mercuri, E.
    Swoboda, K.
    Kernbauer, E.
    Ogrinc, F.
    Menier, M.
    Sproule, D.
    Feltner, D.
    Mendell, J.
    ANNALS OF NEUROLOGY, 2019, 86 : S121 - S121
  • [43] Early intervention and speed-to-effect in spinal muscular atrophy type 1 following onasemnogene abeparvovec gene replacement therapy
    Toro, W.
    Reyna, S.
    Ritter, S.
    Patel, A.
    Mumneh, N.
    Dabbous, O.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S93 - S93
  • [44] Intravenous and intrathecal onasemnogene abeparvovec gene therapy in symptomatic and presymptomatic SMA: long-term follow-up study
    Darras, B.
    Farrar, M.
    Mercuri, E.
    Strauss, K.
    Day, J.
    Chien, Y.
    Masson, R.
    Bernardo, R.
    Alecu, I.
    Dodd, N.
    Mehl, L.
    Connolly, A.
    NEUROMUSCULAR DISORDERS, 2024, 43
  • [45] Onasemnogene abeparvovec gene therapy in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update
    Strauss, K.
    Farrar, M.
    Swoboda, K.
    Saito, K.
    Chiriboga, C.
    Finkel, R.
    Iannaccone, S.
    Krueger, J.
    Kwon, J.
    McMillan, H.
    Servais, L.
    Mendell, J.
    Parsons, J.
    Scoto, M.
    Shieh, P.
    Zaidman, C.
    Schultz, M.
    Ogrinc, F.
    Group, S.
    Muntoni, F.
    NEUROMUSCULAR DISORDERS, 2020, 30 : S122 - S122
  • [46] Can the single intravenous Administration of Gene Replacement Therapy correct the Phenotype in spinal Muscular Atrophy Type 1 (SMA1)?
    Walter, Maggie C.
    FORTSCHRITTE DER NEUROLOGIE PSYCHIATRIE, 2018, 86 (02) : 77 - 78
  • [47] AVXS-101 gene replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal studies clinical update (STR1VE-EU and STR1VE)
    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.
    Schultz, M.
    Shieh, P. B.
    Smith, E. C.
    Feltner, D. E.
    Ouyang, H.
    Lavrov, A.
    Truncated, A.
    L'italien, J.
    Sproule, D. M.
    Mendell, J. R.
    Muntoni, F.
    EUROPEAN JOURNAL OF NEUROLOGY, 2019, 26 : 224 - 225
  • [48] Ethical issues related to gene therapy with onasemnogene abeparvovec for spinal muscular atrophy type 1 in a developing country
    Thong, Meow-Keong
    Tae, Sok-Kun
    Mazlan, Rifhan
    Fong, Choong Yi
    Marie, Nathan Anna
    Li, Limin
    Lim, Wei-Kang
    Eg, Kah-Peng
    GENETICS IN MEDICINE, 2022, 24 (03) : S274 - S275
  • [49] Spinal presentations in children with spinal muscular atrophy type 1 following gene therapy treatment with onasemnogene abeparvovec - The SMA REACH UK network experience
    Wolfe, Amy
    Sheehan, Jennie
    Schofield, Alex
    Cranney, Helen
    O'Reilly, Emer
    Stimpson, Georgia
    Andrews, Alice
    Vanegas, Maria
    Lucas, Jonathan
    Scoto, Mariacristina
    Gowda, Vasantha
    Wraige, Elizabeth
    Jungbluth, Heinz
    NEUROMUSCULAR DISORDERS, 2024, 44
  • [50] THE VALUE OF ONASEMNOGENE ABEPARVOVEC GENE-REPLACEMENT THERAPY IN IMPROVING SURVIVAL AND MOTOR FUNCTION AND DECREASING VENTILATORY SUPPORT AND HOSPITALIZATION CONTRASTED TO NATURAL HISTORY FOR SPINAL MUSCULAR ATROPHY TYPE 1
    Dabbous, O.
    Sproule, D. M.
    Feltner, D. E.
    Ogrinc, F. G.
    Menier, M.
    Droege, M.
    Maru, B.
    Khan, F.
    Arjunji, R.
    VALUE IN HEALTH, 2019, 22 : S841 - S841
12345