Innovative therapeutics


MPS IIIB (Sanfilippo B Syndrome)

Current Clinical Trials: MPS IIIB (Sanfilippo B Syndrome)

Study: A Prospective, Observational Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Indication: MPS IIIB (Sanfilippo B Syndrome)

Investigational Therapeutic: BMN 250

Study Type: Observational

Goal: The purpose of this multicenter, multinational, longitudinal study is to quantify the progression of MPS IIIB over time and to correlate changes in clinical features of the disease, in particular cognitive decline, with both MRI characteristics and biochemical markers of disease burden. This information may help inform the design and interpretation of subsequent interventional studies.

Status: Recruiting

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Study: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular BMN 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Indication: MPS IIIB (Sanfilippo B Syndrome)

Study Type: Phase 1/2

Goal: 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.

Status: Recruiting

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Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI) PKU, LEMS



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