Ionis Pharmaceuticals, Inc.
Compositions and methods for modulating Ataxin 3 expression

Abstract:

Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.