申请人:Indiana University Research and Technology Corporation
公开号:US10420753B2
公开(公告)日:2019-09-24
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.
本文公开了用于治疗脊髓性肌萎缩症(SMA)的组合物和方法。在某些实施方案中,所提供的化合物可增加由 SMN2 基因产生的运动神经元(SMN)全长存活蛋白。