COMPOUNDS FOR TREATING, DELAYING AND/OR PREVENTING A HUMAN GENETIC DISORDER SUCH AS MYOTONIC DYSTROPHY TYPE I (DMI)

摘要

The current invention provides new compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (DM1), spino-cerebellar ataxia 8 and/or Huntington's disease-like 2 caused by expansions of CUG repeats in the transcripts of DM1/DMPK, SCA8 or JPH3 genes.