Sarepta Therapeutics, Inc.
METHODS FOR TREATING MUSCULAR DYSTROPHY WITH CASIMERSEN

Abstract:

The present disclosure provides, among other things, improved compositions and methods for treating muscular dystrophy. For example, the disclosure provides methods for treating Duchenne muscular dystrophy patients having a mutation in the DMD gene that is amenable to exon 45 skipping by administering an effective amount of casimersen.