Event-free survival and motor milestone achievement following onasemnogene abeparvovec and nusinersen interventions contrasted to natural history for spinal muscular atrophy t1 (SMA1) patients

Dabbous O.; Sproule D.; Feltner D.; Ogrinc F.; Menier M.; Droege M.; Maru B.; Khan F.; Arjunji R.

首页> 外文期刊>Neuromuscular disorders: NMD >Event-free survival and motor milestone achievement following onasemnogene abeparvovec and nusinersen interventions contrasted to natural history for spinal muscular atrophy t1 (SMA1) patients

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Event-free survival and motor milestone achievement following onasemnogene abeparvovec and nusinersen interventions contrasted to natural history for spinal muscular atrophy t1 (SMA1) patients

机译：无活动的生存和电机里程碑取得诸如OnAsmaNogene Abeparvovec和Nusinersen干预措施对比脊髓肌肉萎缩T1（SMA1）患者的自然历史形成对比

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《Neuromuscular disorders: NMD》 |2019年第1期|共1页
作者
Dabbous O.; Sproule D.; Feltner D.; Ogrinc F.; Menier M.; Droege M.; Maru B.; Khan F.; Arjunji R.;
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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;

AveXis Inc Bannockburn IL USA;

AveXis Inc Bannockburn IL USA;

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正文语种 eng
中图分类神经病学;
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1. Event-free survival and motor milestone achievement following onasemnogene abeparvovec and nusinersen interventions contrasted to natural history for spinal muscular atrophy t1 (SMA1) patients [J] . Dabbous O., Sproule D., Feltner D., Neuromuscular disorders: NMD . 2019,第Suppla1期

机译：无活动的生存和电机里程碑取得诸如OnAsmaNogene Abeparvovec和Nusinersen干预措施对比脊髓肌肉萎缩T1（SMA1）患者的自然历史形成对比
2. Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy Type 1 (SMA1): Pivotal phase 3 study (STR1VE) update [J] . Day J., Chiriboga C., Crawford T., Neuromuscular disorders: NMD . 2019,第Suppla1期

机译：OnAsmaNogene Abearvovec基因替代疗法（GRT）用于脊柱肌萎缩1型（SMA1）：枢轴相3研究（STR1VE）更新
3. 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
4. Investigating the pre-mRNA splicing of the Survival Motor Neuron genes to model the Spinal Muscular Atrophy disease phenotype. [D] . Gladman, Jordan Tanin. 2010

机译：研究生存运动神经元基因的mRNA前剪接，以建模脊髓性肌萎缩症疾病表型。
5. 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）
6. PND9 EVENT-FREE SURVIVAL AND MOTOR MILESTONE ACHIEVEMENT FOLLOWING AVXS-101 AND NUSINERSEN INTERVENTIONS CONTRASTED TO NATURAL HISTORY FOR TYPE I SPINAL MUSCULAR ATROPHY PATIENTS [O] . O. Dabbous, D.M. Sproule, D.E. Feltner, 2019

机译：PND9无事实的生存和运动里程碑取得的成就后AVXS-101和Nusinersen干预措施与I型脊柱肌萎缩患者的自然历史形成鲜明对比
7. Columbia SMA Project: A Randomized, Control Trial of the Effects of Exercise on Motor Function and Strength in Patients with Spinal Muscular Atrophy (SMA). [R] . De Vivo, D. C. 2012

机译：哥伦比亚sma项目：运动对脊髓性肌萎缩症（sma）患者运动功能和力量影响的随机对照试验。

Event-free survival and motor milestone achievement following onasemnogene abeparvovec and nusinersen interventions contrasted to natural history for spinal muscular atrophy t1 (SMA1) patients

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