Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular BMN 250 in Patients With Mucopolysacch
Brief Summary: The study's primary objectives are to evaluate the safety and tolerability of BMN 250 administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the impact of BMN 250 on cognitive function in patients with MPS IIIB as assessed by the Development Quotient.