Treatment of spinal muscular atrophy in European countries: A call to action.
作者信息
Vázquez-Costa Juan F, Martínez-Moreno Mercedes, Madruga-Garrido Marcos, Munell Francina, Nascimento Andrés, Pitarch-Castellano Inmaculada, Pascual-Pascual Samuel Ignacio, Povedano Mónica, Cattinari Maria Grazia
机构信息
Neuromuscular Diseases Unit, Hospital Universitario y Politécnico La Fe, IIS La Fe, Valencia, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain.
出版信息
Eur J Neurol. 2022 Nov;29(11):3475-3477. doi: 10.1111/ene.15522. Epub 2022 Aug 22.
PMID:35971869
Abstract
摘要
相似文献
1
Treatment of spinal muscular atrophy in European countries: A call to action.
Eur J Neurol. 2022 Nov;29(11):3475-3477. doi: 10.1111/ene.15522. Epub 2022 Aug 22.
2
The emerging spectrum of neurodevelopmental comorbidities in early-onset Spinal Muscular Atrophy.早发型脊髓性肌萎缩症中神经发育合并症的新出现范围。
Eur J Paediatr Neurol. 2024 Jan;48:67-68. doi: 10.1016/j.ejpn.2023.11.006. Epub 2023 Nov 23.
3
Statistical considerations in the study of spinal muscular atrophy. Dallas-Cincinnati-Newington Spinal Muscular Atrophy (DCN-SMA) Study Group.脊髓性肌萎缩症研究中的统计学考量。达拉斯-辛辛那提-纽因顿脊髓性肌萎缩症（DCN-SMA）研究小组。
Muscle Nerve. 1990;13 Suppl:S45-8. doi: 10.1002/mus.880131315.
4
Spinal muscular atrophy - the dawning of a new era.脊髓性肌萎缩症——新时代的曙光
Nat Rev Neurol. 2020 Nov;16(11):593-594. doi: 10.1038/s41582-020-00410-7.
5
Dramatic Innovations in the Treatment of Spinal Muscular Atrophy, But Many Unknowns Remain.脊髓性肌萎缩症治疗取得重大创新，但仍有诸多未知之处。
Pharmacoeconomics. 2022 Apr;40(Suppl 1):3-5. doi: 10.1007/s40273-021-01119-1. Epub 2021 Dec 22.
6
[Expert consensus on rehabilitation management of the spinal muscular atrophy].[脊髓性肌萎缩症康复管理专家共识]
Zhonghua Er Ke Za Zhi. 2022 Sep 2;60(9):883-887. doi: 10.3760/cma.j.cn112140-20220315-00201.
7
Population data on acute infantile and chronic childhood spinal muscular atrophy in Warsaw.
Hum Genet. 1990 Jul;85(2):211-4. doi: 10.1007/BF00193198.
8
Gene therapy for spinal muscular atrophy: hope and caution.脊髓性肌萎缩症的基因治疗：希望与谨慎
Lancet Neurol. 2021 Apr;20(4):251-252. doi: 10.1016/S1474-4422(21)00071-5. Epub 2021 Mar 17.
9
Guidance on Gene Replacement Therapy in Spinal Muscular Atrophy: A Canadian Perspective.脊髓性肌萎缩症基因替代疗法指南：加拿大视角
Can J Neurol Sci. 2022 May;49(3):398-401. doi: 10.1017/cjn.2021.125. Epub 2021 Jun 4.
10
Paradigm shift in the era of disease-modifying therapies for Spinal Muscular Atrophy type 1: respiratory challenges and opportunities.1型脊髓性肌萎缩症疾病修饰疗法时代的范式转变：呼吸方面的挑战与机遇
Sleep Med. 2021 Oct;86:113-115. doi: 10.1016/j.sleep.2021.06.022. Epub 2021 Jun 25.
Zotero 插件