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

Mendell J.; Shell R.; Lehman K.; McColly M.; Lowes L.; Alfano L.; Miller N.; Iammarino M.; Church K.; Ogrinc F.; Ouyang H.; Kernbauer E.; Shah S.; LItalien J.; Sproule D.; Feltner D.; Al-Zaidy S.

首页> 外文期刊>Neuromuscular disorders: NMD >Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial

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

机译：基因替代疗法（GRT）在脊柱肌萎缩1型（SMA1）：来自onAsmnogene abeparvovec期1/2a临床试验的长期随访

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《Neuromuscular disorders: NMD》 |2019年第1期|共1页
作者
Mendell J.; Shell R.; Lehman K.; McColly M.; Lowes L.; Alfano L.; Miller N.; Iammarino M.; Church K.; Ogrinc F.; Ouyang H.; Kernbauer E.; Shah S.; LItalien J.; Sproule D.; Feltner D.; Al-Zaidy S.;
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Nationwide Childrens Hosp Columbus OH USA;

Nationwide Childrens Hosp Columbus OH USA;

Nationwide Childrens Hosp Columbus OH USA;

Nationwide Childrens Hosp Columbus OH USA;

Nationwide Childrens Hosp Columbus OH USA;

Nationwide Childrens Hosp Columbus OH USA;

Nationwide Childrens Hosp Columbus OH USA;

Nationwide Childrens Hosp Columbus OH USA;

Nationwide Childrens Hosp Columbus OH USA;

AveXis Inc Bannockburn IL USA;

AveXis Inc Bannockburn IL USA;

AveXis Inc Bannockburn IL USA;

AveXis Inc Bannockburn IL USA;

AveXis Inc Bannockburn IL USA;

AveXis Inc Bannockburn IL USA;

AveXis Inc Bannockburn IL USA;

Nationwide Childrens Hosp Columbus OH USA;

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正文语种 eng
中图分类神经病学;
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1. Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial [J] . Mendell J., Shell R., Lehman K., Neuromuscular disorders: NMD . 2019,第Suppla1期

机译：基因替代疗法（GRT）在脊柱肌萎缩1型（SMA1）：来自onAsmnogene abeparvovec期1/2a临床试验的长期随访
2. Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG) [J] . Finkel R., Day J., Darras B., Neuromuscular disorders: NMD . 2019,第Suppla1期

机译：用于脊柱肌脂萎缩型2型（SMA2）的鞘内施用onAsmnogene abeparvovec基因替代治疗（GRT）：1/2A研究（强）
3. P.066 AVXS-101 gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the phase 1 clinical trial [J] . JRMendell, KJLehman, MMcColly, The Canadian Journal of Neurological Sciences: le Journal Canadien des Sciences Neurologiques . 2019,第S1期

机译：P.066 AVXS-101基因替代疗法（GRT）在脊柱肌萎缩1型（SMA1）：1阶段临床试验中的长期随访
4. RESULT OF CLINICAL TRIALS WITH CHILDREN WITH SPINAL MUSCULAR ATROPHY USING THE ATLAS 2020 LOWER-LIMB ACTIVE ORTHOSIS [C] . DANIEL SANZ-MERODIO, MANUEL PEREZ, MANUEL PRIETO, International Conference on Climbing and Walking Robots and the Support Technologies for Mobile Machines . 2018

机译：使用ATLAS 2020下肢活性矫形器与脊柱肌萎缩儿童临床试验的结果
5. Glycyl-tRNA synthetase mutations cause Charcot-Marie-Tooth disease type 2D and distal spinal muscular atrophy type V: A potentially novel disease mechanism for human peripheral neuropathies. [D] . Antonellis, Anthony. 2005

机译：糖基-tRNA合成酶突变导致2D型Charcot-Marie-Tooth病和V型远端脊髓性肌萎缩症：人类周围神经病的潜在新型疾病机制。
6. An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER) [O] . Rebecca Dean, Ivar Jensen, Phil Cyr, 2021

机译：脊髓肌萎缩1型患者OnAsemnogene Abeparvovec（Zolgensma®）的更新成本实用新型及临床和有效性评估研究所的评估（ICER）
7. Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial [O] . J.R. Mendell, R. Shell, K.J. Lehman, 2019

机译：脊柱肌肉萎缩（SMA1）中的基因替代疗法（GRT）：来自onAsmnogene abeparvovec期1/2a临床试验的长期随访
8. Tritherapy (Spinalon)-Elicited Spinal Locomotor Network Activation: Phase I-IIa Clinical Trial in Spinal Cord-Injured Patients. [R] . Guertin, P., Vaillancourt, M. 2013

机译：Tritherapy（spinalon） - 激活脊髓运动网络激活：脊髓损伤患者的I-IIa期临床试验。

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

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