别名 ALS、ALS、ALS (amyotrophic lateral sclerosis) + [83] |
简介 A degenerative disorder affecting upper MOTOR NEURONS in the brain and lower motor neurons in the brain stem and SPINAL CORD. Disease onset is usually after the age of 50 and the process is usually fatal within 3 to 6 years. Clinical manifestations include progressive weakness, atrophy, FASCICULATION, hyperreflexia, DYSARTHRIA, dysphagia, and eventual paralysis of respiratory function. Pathologic features include the replacement of motor neurons with fibrous ASTROCYTES and atrophy of anterior SPINAL NERVE ROOTS and corticospinal tracts. (From Adams et al., Principles of Neurology, 6th ed, pp1089-94) |
作用机制 TFE3 agonists [+3] |
最高研发阶段批准上市 |
首次获批国家/地区 美国 |
首次获批日期2024-09-20 |
靶点 |
作用机制 ETB激动剂 [+3] |
在研机构 |
最高研发阶段批准上市 |
首次获批国家/地区 印度 |
首次获批日期2023-05-31 |
开始日期2025-12-01 |
申办/合作机构 |
开始日期2025-10-01 |
申办/合作机构 |
开始日期2025-09-01 |