JR-446 clinical trials

A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

This study is looking into a new medicine called JR-446 for young children (under 6) with a rare genetic condition called Mucopolysaccharidosis Type IIIB (MPS IIIB). We want to see if the medicine is safe and helps with the condition.