PTC Therapeutics, Inc.
THIOENO[3,2-B] PYRIDIN-7-AMINE COMPOUNDS FOR TREATING FAMILIAL DYSAUTONOMIA

Abstract:

The present description relates' to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia. ##STR00001##