PETER CHRISTIAN DE VISSER;MARIA BEGONA AGUILERA DIEZ;SUSAN ALLEGONDA MARIA MULDERS;PROSENSA TECHNOLOGIES B.V.
发明人:
申请号：
IL22902213
公开号：
IL229022D0
申请日:
2013.10.22
申请国别(地区):
IL
年份:
2013
代理人:
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.
