3Rd Neurology Unit and Motor Neuron Disease Centre, Fondazione IRCCS "Carlo Besta" Neurological Insitute, Milan, Italy.
Experimental Neuropathology Unit, Division of Neuroscience, Institute of Experimental Neurology, IRCCS San Raffaele Scientific Institute, Milan, Italy.
J Neurol. 2024 Jul;271(7):4693-4723. doi: 10.1007/s00415-024-12435-9. Epub 2024 May 27.
In the last few years, our understanding of disease molecular mechanisms underpinning ALS has advanced greatly, allowing the first steps in translating into clinical practice novel research findings, including gene therapy approaches. Similarly, the recent advent of assistive technologies has greatly improved the possibility of a more personalized approach to supportive and symptomatic care, in the context of an increasingly complex multidisciplinary line of actions, which remains the cornerstone of ALS management. Against this rapidly growing background, here we provide an comprehensive update on the most recent studies that have contributed towards our understanding of ALS pathogenesis, the latest results from clinical trials as well as the future directions for improving the clinical management of ALS patients.
在过去的几年中,我们对 ALS 疾病分子机制的理解有了很大的进展,使得将新的研究发现转化为临床实践的第一步成为可能,包括基因治疗方法。同样,最近辅助技术的出现极大地提高了在日益复杂的多学科行动线的背景下,对支持性和症状性护理进行更个性化的方法的可能性,这仍然是 ALS 管理的基石。在这一快速发展的背景下,我们在这里提供了对 ALS 发病机制的最新研究、临床试验的最新结果以及改善 ALS 患者临床管理的未来方向的最新研究的综合更新。