Inventors:
- La Jolla CA, US
- San Diego CA, US
Suzanne Rzuczek - Jupiter FL, US
John Ndungu - West Palm Beach FL, US
Joseph Vacca - Telford PA, US
Andy Jennings - San Diego CA, US
Alexei Pushechnikov - San Diego CA, US
International Classification:
C07D 235/20
A61P 25/14
C07D 407/14
A61P 25/28
A61P 21/00
Abstract:
Provided herein are compounds and methods for modulating abnormal repeat expansions of gene sequences. More particularly, provided are inhibitors of RNA and the uses of such inhibitors in regulating nucleotide repeat expansions, e.g., to treat Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2 (DM2), Fuchs dystrophy, Huntington Disease, Amyotrophic Lateral Sclerosis, or Frontotemporal Dementia.