A Treatment Extension Study of Mucopolysaccharidosis Type IIIB
Status:
Active, not recruiting
Trial end date:
2025-02-01
Target enrollment:
Participant gender:
Summary
The primary objectives of this study are to evaluate the long-term safety and tolerability of
AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and
to evaluate the impact of long-term AX 250 treatment on cognitive function in patients with
MPS IIIB as assessed by developmental quotient (DQ).