21:21 , Nov 10, 2017 |  BC Week In Review  |  Clinical News

BioMarin reports Phase I/II data for BMN 250 in mucopolysaccharidosis IIIB

In September, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported interim data from 3 patients with mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo type B syndrome) in the dose-escalation portion of the Phase I/II BMN 250-201 trial showing that intracerebroventricular...
07:00 , May 2, 2016 |  BC Week In Review  |  Clinical News

BMN 250: Phase I/II started

BioMarin began the open-label, dose-escalation, international Phase I/II BMN 250-201 trial to evaluate BMN 250 given via intracerebroventricular infusion. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), San Rafael, Calif.   Product: BMN 250   Business: Endocrine/Metabolic   Molecular...
07:00 , Oct 16, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Endocrine/metabolic disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Endocrine/metabolic disease Mucopolysaccharidosis N-acetylglucosaminidase-a (NAGLU); insulin-like growth factor-2 (IGF-2); mannose-6-phosphate receptor (M6P receptor) Mouse studies suggest a NAGLU-IGF-2 fusion protein could help treat mucopolysaccharidosis IIIB and...