Current and emerging treatment options for spinal muscular atrophy

Faraz Farooq; Alex E MacKenzie

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Current and emerging treatment options for spinal muscular atrophy

机译：脊柱肌肉萎缩的当前和新兴治疗选择

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Abstract: Spinal muscular atrophy is one of the most common inherited neuromuscular conditions; our understanding of the genetic pathology and translational research coming from this insight has made significant progress over the past decade. This short review provides the background of the disease along with the bench to bedside progress of some promising treatment options to develop better understanding of the present state of the disease.

机译：【摘要】脊髓性肌萎缩是最常见的遗传性神经肌肉疾病之一。在过去的十年中，我们对遗传病理学和转化研究的了解取得了重大进展。这篇简短的综述提供了该疾病的背景，以及一些有前途的治疗选择的从实验台到床边的进展，以加深对疾病当前状态的了解。

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《Degenerative Neurological and Neuromuscular Disease》 |2015年第557期|共7页
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Faraz Farooq; Alex E MacKenzie;
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中图分类神经病学;
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机译：目前对呼吸窘迫型脊髓肌萎缩的良好治疗选择1（Smard1）
2. Nucleic Acid–Targeted Small Molecules have Therapeutic Potential in the Treatment of Spinal Muscular Atrophy: Small‐molecule drugs that can selectively bind RNA and modulate pre‐mRNA splicing have potential as a treatment strategy for human disease, including spinal muscular atrophy [J] . American journal of medical genetics, Part A . 2018,第8期

机译：核酸靶心的小分子在治疗脊柱肌萎缩中具有治疗潜力：可以选择性地结合RNA并调节前mRNA剪接的小分子药物作为人类疾病的治疗策略，包括脊柱肌肉萎缩
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4. A Motor Imagery-based Brain-Computer Interface Scheme for a Spinal Muscular Atrophy Subject in CYBATHLON Race [C] . Shi-Chun Bao, Kai Yuan, Cheng Chen, International IEEE/EMBS Conference on Neural Engineering . 2021

机译：基于电机图像的脑电电脑界面方案，用于Cybathlon Race中的脊柱肌肉萎缩课程
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. Current and emerging treatment options for spinal muscular atrophy [O] . Faraz Farooq, Alex E MacKenzie 2015

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7. Current and future treatments of spinal muscular atrophy [O] . Martin Smith 2019

机译：脊髓肌肉萎缩的当前和未来治疗

Current and emerging treatment options for spinal muscular atrophy

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