IDefine is committed to identifying life-changing treatments & cures for those with rare genetic disorders like Kleefstra Syndrome (KS), while building community for families. Our initial concentration is centered around KS, a rare genetic disorder caused by a mutation or deletion of the EHMT1 gene. In consultation with our Scientific Advisory Board, we are pursuing the most promising avenues for drug development, and will "go where the science takes us". A range of approaches are being reviewed, including small-molecule drug repurposing, small molecule drug development, oligonucleotide-based therapies, and gene therapies.