A Treatment Study of Mucopolysaccharidosis Type IIIB
Status:
Completed
Trial end date:
2020-07-31
Target enrollment:
Participant gender:
Summary
The study's primary objectives are to evaluate the safety and tolerability of AX 250
administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the
impact of AX 250 on cognitive function in patients with MPS IIIB as assessed by the
Development Quotient.