Sixth Neurology Department (Department of Neuroinfectious Diseases), Research Center of Neurology, Moscow, Russia.
Med Res Rev. 2021 Sep;41(5):2634-2655. doi: 10.1002/med.21705. Epub 2020 Jul 8.
Motor neuron disorders are a group of neurodegenerative diseases characterized by muscle weakness, loss of ambulation, respiratory insufficiency, leading to an early death. Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis are the most common and fatal motor neuron diseases. The last 3 years became very successful for novel gene therapy approaches in SMA in infants. Two innovative drugs-nusinersen (Spinraza) and onasemnogene abeparvovec (Zolgensma) have been approved by health authorities. The numerous molecular and genetic overlaps between different neurodegenerative diseases are of great importance in the development of innovative therapeutic strategies, including viral vector therapy and RNA modulating approaches.
运动神经元疾病是一组神经退行性疾病，其特征是肌肉无力、行走能力丧失、呼吸功能不全，导致早逝。脊髓性肌萎缩症（SMA）和肌萎缩侧索硬化症是最常见和最致命的运动神经元疾病。过去 3 年，婴儿期脊髓性肌萎缩症的新型基因治疗方法取得了巨大成功。两种创新药物——nusinersen（Spinraza）和onasemnogene abeparvovec（Zolgensma）已获得卫生部门的批准。不同神经退行性疾病之间存在许多分子和遗传重叠，这对于开发创新的治疗策略非常重要，包括病毒载体治疗和 RNA 调节方法。
