Dr. Jacob at Tufts University has 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 our patients. One version has already been through phase II clinical trials for a different disorder here 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.
Will you help us?
This groundbreaking progress is supported by our generous donors. Please donate today to keep moving us closer to a better life for our loved ones with CTNNB1 syndrome.
