abstract |
Compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in cells or animals, and in certain cases for reducing the amount of ataxin-2 protein in cells or animals are provided. The compounds, methods, and pharmaceutical compositions are useful for ameliorating one or more signs or characteristics of a neurodegenerative disease. Such signs and features include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include spinal cerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and Parkinsonism. |