Nafisa Nuzhat, PhD, and Matthew Kneusel, PhD, looking at Western blot results.
Receiving care from a specialist allows individuals to receive specific interventions to improve their quality of life. Non-pharmaceutical interventions like therapy help improve the quality of life for some patients, but often, patients benefit most from drug treatment. The Kodani lab uses genetic and molecular information to identify potential drug treatments (including clinical trials) for patients with rare diseases. Sometimes, that is as simple as identifying an existing drug that will help the patient. By screen- ing hundreds of thousands of drugs in publicly available drug libraries, Kodani and his lab try to find potential compounds that will help re-establish the signaling pathways that went awry in neuro- developmental diseases. Their ultimate goal is to repurpose FDA-approved drugs for use in patients with rare diseases. In other cases, the scientists get creative and look toward natural compounds or dietary changes that could modulate the broken pathway, thus improving the quality of life in indi- viduals with neurodevelopmental disorders. ■
I truly believe that every child deserves a happy childhood. I know we can’t fix every disorder, but we won’t let families continue to face uncertainty alone.
—ANDREW KODANI, PHD
RESEARCH REPORT 2023-2024
21
Made with FlippingBook - Online magazine maker