ARTICLE | Distillery Therapeutics
Therapeutics: Inhibitor of κ light polypeptide gene enhancer in B-cells kinase complex-associated protein (IKBKAP)
March 5, 2015 8:00 AM UTC
In vitro studies identified a compound that could help treat familial dysautonomia, which is often caused by an IKBKAP mutation that results in skipping of exon 20 and consequently misspliced protein....