Nizubaglustat clinical trials

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

This study looks at a new drug, Nizubaglustat, for Niemann-Pick Type C (NPC) disease in children and young people. It aims to see if the drug can help with movement problems and if it's safe, compared to a dummy pill.