BioMarin Provides Program Update on Vosoritide in Achondroplasia
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BioMarin Pharma (BMRN) Announces Enrollment of First BMN 250 Phase 1/2 Patient for MPS IIIB
April 21, 2016 4:34 PM EDTBioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that it has enrolled the first patient in a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB). Discovered by BioMarin, BMN 250 is being studied in a multicenter, international clinical trial evaluating safety and tolerability, as well as cognitive function of patients with MPS IIIB receiving BMN 250. Designed to restore functional NAGLU activity in the brain, BMN 250 is administered via... More
