Donate for Small Molecule Research
CTNNB1 Connect and Cure and Dr. Jacob at Tufts University in the United States have identified a small molecule that shows the potential to alleviate many of our dragonfly’s learning and motor symptoms. She has been working diligently with medicinal chemists to find the best version of this small molecule to use in patients. One version has already been through phase II clinical trials for a different disorder in the United States! Six CTNNB1 patient-derived cell lines from Simons Searchlight are currently being tested, which will help determine which version to move forward with.