National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
Departments of Neurology and Psychiatry, Uniformed Services University of the Health Sciences, Bethesda, MD, USA.
Amyotroph Lateral Scler Frontotemporal Degener. 2020 Nov;21(sup1):67-73. doi: 10.1080/21678421.2020.1837180.
Primary lateral sclerosis (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.
原发性侧索硬化症（PLS）是一种极为罕见的中枢神经系统退行性疾病，其特征是运动神经元进行性缓慢丧失，导致严重的肢体和延髓功能障碍和残疾。虽然不一定会缩短寿命，但 PLS 的疾病负担很大，改善症状治疗是一个主要的未满足需求，特别是对于该综合征的核心特征——通常难以治疗的痉挛。在第 1 节中，我们描述了临床护理需求，并强调了一种高度个性化的方法，这种方法可以通过多学科管理来实现。在第 2 节中，我们描述了 PLS 临床试验的进展，包括症状治疗、疾病修饰治疗和新兴创新试验方面的进展。
