MPS III B research hub

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

This study is an ongoing look at a treatment called AX 250 for MPS IIIB, a rare genetic condition. It's for people who have already been taking AX 250 for a while. Doctors want to see if it's safe and helps with thinking, communication, and daily life over a longer period.