abstract |
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. |