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Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy

机译：新型皮下鞘内导管系统对儿童和成人脊髓性肌萎缩症反复门诊应用Nusinersen的初步安全性和耐受性

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Background:Many patients with spinal muscular atrophy (SMA) who might benefit from intrathecal antisense oligonucleotide (nusinersen) therapy have scoliosis or spinal fusion that precludes safe drug delivery. To circumvent spinal pathology, we designed a novel subcutaneous intrathecal catheter (SIC) system by connecting an intrathecal catheter to an implantable infusion port.

机译：背景：许多可能会从鞘内反义寡核苷酸（nusinersen）治疗中受益的脊髓性肌萎缩症（SMA）患者患有脊柱侧弯或脊柱融合术，从而无法安全地给药。为了规避脊柱病理，我们通过将鞘内导管连接到可植入输液端口来设计新型皮下鞘内导管（SIC）系统。

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期刊名称 Lippincott Williams Wilkins Open Access
作者
Kevin A. Strauss; Vincent J. Carson; Karlla W. Brigatti; Millie Young; Donna L. Robinson; Christine Hendrickson; Michael D. Fox; Robert M. Reed; Erik G. Puffenberger; William Mackenzie; Freeman Miller;
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年(卷),期 -1(38),10
年度 -1
页码 e610–e617
总页数 8
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1. Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy [J] . Strauss Kevin A., Carson Vincent J., Brigatti Karlla W., Journal of pediatric orthopaedics . 2018,第10期

机译：一种新型皮下鞘内导管系统的初步安全性和耐受性，用于脊髓肌萎缩的儿童和成人的重复门诊室外剂量
2. A 5-center experience with intrathecal administration of nusinersen in SMA1 in Italy letter to the editor of european journal of pediatric neurology regarding the manuscript "single-center experience with intrathecal administration of nusinersen in children with spinal muscular atrophy type 1" written by pechmann and colleagues" [J] . Sansone V. A., Pane M., Messina S., European journal of paediatric neurology: EJPN : official journal of the European Paediatric Neurology Society . 2018,第4期

机译：在意大利SMA1中的SMA1中努力施用的5中心经验，致欧洲小儿科杂志的编辑，关于手稿“单中心经验，鞘内经历血液肌肉萎缩1型儿童血液肌肉萎缩1型儿童血液肌肉萎缩1型”和Pechmann和同事”
3. Computed‐tomography‐guided transforaminal intrathecal nusinersen injection in adults with spinal muscular atrophy type 2 and severe spinal deformity. Feasibility, safety and radiation exposure considerations [J] . Spiliopoulos S., Reppas L., Zompola C., European journal of neurology: the official journal of the European Federation of Neurological Societies . 2020,第7期

机译：计算断层扫描引导的晶状体鞘内切除鼻内鼻腔注射液，脊髓肌萎缩2型和严重脊柱畸形。可行性，安全性和辐射照射注意事项
4. EXOtrainer Project Clinical Evaluation of Gait Training with Exoskeleton in Children with Spinal Muscular Atrophy [C] . Daniel Sanz-Merodio, Gonzalo Puyuelo, Amartya Ganguly, International Conference on Intelligent Robots . 2020

机译：Exotrainer在脊髓肌肉萎缩儿童中对外骨骼进行步态训练的临床评价
5. Nusinersen Treatment and Healthcare Costs in Spinal Muscular Atrophy [D] . Fox, David A. 2020

机译：血缘肌肉萎缩的螺母治疗和医疗保健成本
6. Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy [O] . Benjamin Stolte, Andreas Totzeck, Kathrin Kizina, 2018

机译：成年神经鞘内鞘内注射治疗成人脊髓性肌萎缩症的可行性和安全性
7. Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy [O] . Kevin A. Strauss, Vincent J. Carson, Karlla W. Brigatti, 2018

机译：一种新型皮下鞘内导管系统的初步安全性和可耐受性，用于脊髓肌萎缩的儿童和成人的重复门诊室外剂量

Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy

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