Clinical care and therapeutic trials in PLS.

Clinical care and therapeutic trials in PLS.
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DOI:
10.1080/21678421.2020.1837180
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发表时间:
2020-11
影响因子:
2.8
通讯作者:
Mitsumoto H
Mitsumoto H
中科院分区:
医学4区
文献类型:
--
作者:
Floeter MK;Warden D;Lange D;Wymer J;Paganoni S;Mitsumoto H

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PLS是一种极为罕见的中枢神经系统退行性疾病,其特征是缓慢进行性上运动神经元丢失,导致严重的肢体和延髓功能障碍和残疾。尽管不一定会缩短寿命,但PLS疾病负担很大,改进的对症治疗是一个主要未满足的需求,特别是对于作为该综合征核心特征的难治性痉挛。在第1节中,我们描述了临床护理需求,并强调了高度个性化的方法,可以通过多学科管理最好地实现。在第2节中,我们描述了PLS临床试验的进展,包括对症治疗,疾病修饰治疗和新兴创新试验的进展。
PLS is an extremely rare central nervous system degenerative disorder characterized by slowly progressive upper motor neuron loss leading to severe limb and bulbar dysfunction and disability. Although not necessarily life-shortening, PLS disease burden is substantial and improved symptomatic treatments are a major unmet need, especially for the often refractory spasticity that is a core feature of the syndrome. In Section 1, we describe clinical care needs and emphasize a highly personalized approach that can be best attained through multidisciplinary management. In Section 2, we describe progress in clinical trials in PLS that includes advances in symptomatic treatment, disease-modifying therapy, and emerging innovative trials.
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