Compositions including small molecule biochemicals, and salts and derivatives thereof, and methods for detection, treatment or prophylaxis of neuromuscular disorders are provided, including modulation of microRNA that contribute to neuromuscular disorders, including muscular dystrophies, peripheral motor neuron diseases, motor neuron diseases, neuromuscular junction diseases, myopathies, metabolic diseases of the muscle, amyotrophic lateral sclerosis, multiple sclerosis, Parkinson's disease, motor neuron diseases, Huntington's disease, spinocerebellar ataxia, and spinal muscular atrophy.
提供了包括小分子生化物质及其盐和衍
生物在内的组合物,以及用于检测、治疗或预防神经肌肉疾病的方法,包括调节导致神经肌肉疾病(包括肌肉萎缩症)的微RNA、外周运动神经元疾病、运动神经元疾病、神经肌肉接头疾病、肌病、肌肉代谢疾病、肌萎缩侧索硬化症、多发性硬化症、帕
金森病、运动神经元疾病、亨廷顿病、脊髓小脑共济失调症和脊髓性肌萎缩症。