COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT
摘要
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
申请公布号
EP2442816(A4)
申请公布日期
2013.10.02
申请号
EP20100790221
申请日期
2010.06.17
申请人
ISIS PHARMACEUTICALS, INC.;COLD SPRING HARBOR LABORATORY
