NGLY Collaboration & Discovery Efforts

We are collaborating with the National Center for Advancing Translational Sciences (NCATS) Therapeutics for Rare and Neglected Diseases (TRND) Biology Laboratory, and patient advocacy foundation on a research plan to develop different assays for small molecule high-throughput screening. This is being done in both an effort to better understand the biology of the disease, as well as identify potential small molecules to be developed as a therapeutic for patients suffering from NGLY Deficiency.