Azafaros A.g.

This study tests the long-term safety and effects of a daily medication called nizubaglustat in people with two rare genetic brain diseases: GM2 gangliosidosis and Niemann-Pick type C. About 21 participants who were in a previous study or who are already on a similar drug will ta…

This study tests an oral drug called nizubaglustat in children and teens aged 4 and older with rare genetic brain diseases: Niemann-Pick type C, GM1, or GM2 gangliosidosis. The drug is taken by mouth for 18 months and compared to a placebo. The goal is to see if it can safely slo…

This study tests an oral medication called nizubaglustat in children aged 4 and older with Niemann-Pick type C disease, a rare genetic disorder that affects movement and thinking. The trial lasts 18 months and compares the drug to a placebo to see if it can slow worsening of coor…

This study tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with GM1 or GM2 gangliosidosis—rare genetic disorders that damage the brain and nerves. Over 18 months, half the participants will receive the drug and half a placebo to see if it slows diseas…