最近关于脊髓性肌萎缩症治疗的研究。
Recent research on the treatment of spinal muscular atrophy.
机构信息
School of Public Health and Management, Guangxi University of Chinese Medicine, Nanning 530022, China.
出版信息
Zhongguo Dang Dai Er Ke Za Zhi. 2022 Feb 15;24(2):204-209. doi: 10.7499/j.issn.1008-8830.2110041.
Abstract
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by progressive muscular weakness and atrophy. SMA, as an inherited disease, is the leading cause of death in infants and young children. Rapid progress has been made in the research field of SMA in recent years, and some related treatment drugs have been successfully approved for marketing. This article reviews the recent research advances in the treatment of SMA.
摘要
脊髓性肌萎缩症（SMA）是一种常染色体隐性神经肌肉疾病，其特征是进行性肌肉无力和萎缩。SMA 作为一种遗传病，是婴儿和幼儿死亡的主要原因。近年来，SMA 研究领域取得了快速进展，一些相关的治疗药物已成功获准上市。本文综述了 SMA 治疗的最新研究进展。
相似文献
1
Recent research on the treatment of spinal muscular atrophy.
Zhongguo Dang Dai Er Ke Za Zhi. 2022 Feb 15;24(2):204-209. doi: 10.7499/j.issn.1008-8830.2110041.
2
Established Stem Cell Model of Spinal Muscular Atrophy Is Applicable in the Evaluation of the Efficacy of Thyrotropin-Releasing Hormone Analog.
Stem Cells Transl Med. 2016 Feb;5(2):152-63. doi: 10.5966/sctm.2015-0059. Epub 2015 Dec 18.
3
[Risdiplam for the treatment of spinal muscular atrophy].
Zh Nevrol Psikhiatr Im S S Korsakova. 2024;124(2):45-57. doi: 10.17116/jnevro202412402145.
4
The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy.
Neuromuscul Disord. 2019 Jan;29(1):21-29. doi: 10.1016/j.nmd.2018.10.001. Epub 2018 Oct 30.
5
Nusinersen for the treatment of spinal muscular atrophy.
Expert Rev Neurother. 2017 Oct;17(10):955-962. doi: 10.1080/14737175.2017.1364159. Epub 2017 Sep 8.
6
Spinal muscular atrophies.
Pediatr Clin North Am. 2015 Jun;62(3):743-66. doi: 10.1016/j.pcl.2015.03.010. Epub 2015 Apr 11.
7
Spinal Muscular Atrophy Treatment: The MTOR Regulatory Intervention.
Curr Med Chem. 2024;31(12):1512-1522. doi: 10.2174/0929867330666230213114909.
8
Current advances in drug development in spinal muscular atrophy.
Curr Opin Pediatr. 2013 Dec;25(6):682-8. doi: 10.1097/MOP.0b013e32836565ac.
9
Advances and limitations for the treatment of spinal muscular atrophy.
BMC Pediatr. 2022 Nov 3;22(1):632. doi: 10.1186/s12887-022-03671-x.
10
Spinal Muscular Atrophy and Common Therapeutic Advances.
Fetal Pediatr Pathol. 2019 Jun;38(3):226-238. doi: 10.1080/15513815.2018.1520374. Epub 2019 May 7.
本文引用的文献
1
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9.
2
Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1.
Muscle Nerve. 2021 Oct;64(4):487-490. doi: 10.1002/mus.27375. Epub 2021 Jul 31.
3
A Randomized Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Study of the Novel Myostatin Inhibitor Apitegromab (SRK-015): A Potential Treatment for Spinal Muscular Atrophy.
Adv Ther. 2021 Jun;38(6):3203-3222. doi: 10.1007/s12325-021-01757-z. Epub 2021 May 8.
4
Prenatal transplantation of human amniotic fluid stem cell could improve clinical outcome of type III spinal muscular atrophy in mice.
Sci Rep. 2021 Apr 28;11(1):9158. doi: 10.1038/s41598-021-88559-z.
5
Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study.
Neurotherapeutics. 2021 Apr;18(2):1127-1136. doi: 10.1007/s13311-020-01004-3. Epub 2021 Feb 23.
6
Multidisciplinary approach and psychosocial management of spinal muscular atrophy (SMA).
Arch Pediatr. 2020 Dec;27(7S):7S45-7S49. doi: 10.1016/S0929-693X(20)30277-3.
7
Risdiplam: First Approval.
Drugs. 2020 Nov;80(17):1853-1858. doi: 10.1007/s40265-020-01410-z.
8
Combination molecular therapies for type 1 spinal muscular atrophy.
Muscle Nerve. 2020 Oct;62(4):550-554. doi: 10.1002/mus.27034. Epub 2020 Aug 10.
9
New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?
Int J Mol Sci. 2020 May 7;21(9):3297. doi: 10.3390/ijms21093297.
10
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.
Neuromuscul Disord. 2019 Nov;29(11):842-856. doi: 10.1016/j.nmd.2019.09.007. Epub 2019 Sep 12.
Zotero 插件