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Mar 05, 2015
 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Inhibitor of κ light polypeptide gene enhancer in B-cells kinase complex-associated protein (IKBKAP)

Neurology

INDICATION: Neuropathy

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....

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