Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disease that targets motor neurons (MNs) in the brain and spinal cord. It leads to gradual loss of motor signals to muscles leading to atrophy and weakness. Most patients do not survive for more than 3–5 years after disease onset. Current ALS treatments provide only a small delay of disease progression. Therefore, it is of utmost importance to explore new therapeutic approaches. One of the major hindrances in achieving this goal is poor understanding of causes of the disease. ALS has complex pathophysiological mechanisms in its genetic and sporadic forms. Protein aggregates are a common hallmark of ALS regardless of cause making protein pathways attractive therapeutic targets in ALS. Here, we provide an overview of compounds in different stages of pharmacological development and their protein pathway targets.

肌萎缩性侧索硬化症（ALS）是一种以大脑和脊髓中的运动神经元（MNs）为目标的虚弱性神经退行性疾病。它导致肌肉运动信号逐渐丢失，导致萎缩和无力。大多数患者在疾病发作后不能存活超过3-5年。当前的ALS治疗仅提供疾病进展的小延迟。因此，探索新的治疗方法至关重要。实现这一目标的主要障碍之一是对疾病原因的了解不足。ALS以其遗传形式和散发形式具有复杂的病理生理机制。蛋白质聚集体是ALS的常见标志，无论其原因如何使蛋白质途径成为ALS中有吸引力的治疗靶标。这里，