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MPS IIIB (Sanfilippo B Syndrome)

Current Clinical Trials: MPS IIIB (Sanfilippo B Syndrome)

Study 250-901: A Prospective, Observational Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Indication: MPS IIIB (Sanfilippo B Syndrome)

Investigational Therapeutic: Tralesinidase alfa (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: Active, not recruiting

Additional information: View on Clinicaltrials.gov


Study 250-201: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular Tralesinidase alfa (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 tralesinidase alfa (BMN 250) administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the impact of tralesinidase alfa (BMN 250) on cognitive function in patients with MPS IIIB as assessed by the Development Quotient.

Status: Recruiting

Additional information: View on Clinicaltrials.gov


Study 250-902: A Prospective Natural History Study of Mucopolysaccharidosis
Type IIIB (MPS IIIB)

Indication: Mucopolysaccharidosis Type IIIB (MPS IIIB)

Study Type: Prospective, non-interventional, natural history

Goal: To quantify the progression of cognitive decline in pediatric patients with MPS IIIB over time. This information may help inform the design and interpretation of interventional studies in this MPS IIIB population.

Status: Recruiting

Additional information: View on Clinicaltrials.gov

With your help, researchers can better understand the progression of MPS and related diseases

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BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

PALYNZIQ
Brineura
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
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Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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