Niemann-Pick Type C Disease research hub

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, GM1 Gangliosidosis or GM2 Gangliosidosis

This study is looking at a new medicine called AZ-3102 for children and young people with rare conditions like Niemann-Pick Type C, GM1 Gangliosidosis, or GM2 Gangliosidosis. It aims to see if the medicine is safe and if it helps improve their health over 18 months.