David W. Piotrowski

Discovery of Neutral and Cerebrospinal Fluid Penetrant Transthyretin (TTR) Stabilizers with Heterocyclic Linkers

Abstract Human transthyretin (TTR) is associated with ATTR amyloidosis. Despite having approved agents against TTR, there is still an unmet medical need to develop novel agents, especially those that could have therapeutic effects in cerebrospinal fluid (CSF). Structure-based dru...