PTC Therapeutics, Inc.
PYRROLO[2,3-D]PYRIMIDINE 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 described herein relates to substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia. ##STR00001##