Last updated on September 2018

A Treatment Study of Mucopolysaccharidosis Type IIIB


Brief description of study

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.

Clinical Study Identifier: NCT02754076

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