Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)

Schultz M.; Swoboda K.; Farrar M.; McMillan H.; Parsons J.; Kernbauer E.; Farrow M.; Ogrinc F.; Kavanagh S.; Feltner D.; McGill B.; Spector S.; LItalien J.; Sproule D.; Strauss K.

首页> 外文期刊>Neuromuscular disorders: NMD >Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)

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Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)

机译：在症状脊髓肌萎缩（SMA）中onAnaMeNogene Abeparvovec基因替代疗法（GRT）

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来源
《Neuromuscular disorders: NMD》 |2019年第1期|共1页
作者
Schultz M.; Swoboda K.; Farrar M.; McMillan H.; Parsons J.; Kernbauer E.; Farrow M.; Ogrinc F.; Kavanagh S.; Feltner D.; McGill B.; Spector S.; LItalien J.; Sproule D.; Strauss K.;
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作者单位

AveXis Inc Bannockburn IL USA;

Massachusetts Gen Hosp Boston MA USA;

Sydney Childrens Hosp Randwick NSW Australia;

Canada Childrens Hosp Ottawa ON Canada;

Childrens Hosp Colorado Aurora CO 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;

AveXis Inc Bannockburn IL USA;

Clin Special Children Strasburg PA USA;

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正文语种 eng
中图分类神经病学;
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专利

1. Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA) [J] . Schultz M., Swoboda K., Farrar M., Neuromuscular disorders: NMD . 2019,第Suppla1期

机译：在症状脊髓肌萎缩（SMA）中onAnaMeNogene Abeparvovec基因替代疗法（GRT）
2. 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
3. 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）
4. Isokinetic test and exercise in spinal muscular atrophy (SMA) [C] . Granata C., Colombari M., Ballestrazzi A., Engineering in Medicine and Biology Society, 1989. Images of the Twenty-First Century., Proceedings of the Annual International Conference of the IEEE Engineering in . 1989

机译：脊髓性肌萎缩症（SMA）的等速测试和运动
5. Spinal Muscular Atrophy (SMA): The Newborn and Carrier Screening Debate [D] . Ehivet, Fabienne Estelle 2012

机译：脊髓性肌萎缩症（SMA）：新生儿和携带者筛查辩论
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. Onasemnogene abeparvovec gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update [O] . K.A. Strauss, K.J. Swoboda, M.A. Farrar, 2019

机译：在假设脊髓肌萎缩（SMA）中的OnAsmaNogene Abearvovec基因替代疗法（GRT）：SPR1NT研究更新
8. 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。

Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)

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