BioMarin Pharmaceutical has initiated a Phase I/II trial to evaluate BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase, as a treatment for Pompe disease.
The open-label study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamic and clinical activity of BMN 701, which will be administered as an intravenous infusion every two weeks at doses of 5mg/kg, 10mg/kg and 20mg/kg.
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BioMarin plans to enrol approximately 30 patients aged 13-65 years old with late-onset Pompe disease for a treatment period of 24 weeks.
Pompe disease, a lysosomal storage disorder, is a progressive degenerative muscle disease that occurs in one in every 40,000 births.
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