Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.
本文揭示了治疗脊髓性肌萎缩症(SMA)的组合物和方法。在某些实施例中,提供了一些化合物,可以通过SMN2
基因增加全长生存运动神经元(SMN)蛋白的产生。