The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N—((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.
本公开内容提供了治疗或预防肌萎缩性脊髓侧索硬化症(ALS)的新方法、延缓与ALS相关的神经症状发作的方法、提高ALS患者存活率的方法,以及减轻有需要的受试者与ALS相关的肌力下降的方法。本公开还提供了治疗或预防α-突触核蛋白病或TDP-43蛋白病的方法。这些方法包括向受试者施用有效量的线粒体靶向拟肽化合物,如(R)-2-
氨基-N-((S)-1-((S)-5-
氨基-1-(3-苄基-1,2、4-恶二唑-5-基)戊基)
氨基)-3-(
4-羟基-2,6-二甲基苯基)-1-氧代
丙烷-2-基)-5-
胍基戊酰胺,或其药学上可接受的盐、立体异构体、同分异构体、
水合物和/或溶液。