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.