BMRN
- BioMarin Pharmaceutical Inc.
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Overview
Company Summary
BioMarin Pharmaceutical Inc. (BMRN) is a biotechnology company that specializes in developing and commercializing innovative therapies for rare genetic diseases. The company's primary focus is on diseases that have limited treatment options and affect a small patient population.
BioMarin is dedicated to applying advanced genetic science to develop therapies that address the underlying cause of these rare diseases, rather than just managing their symptoms. They utilize cutting-edge technologies, such as gene therapy, enzyme replacement therapy, and gene editing, to develop potential treatments.
BioMarin's product portfolio includes a range of therapies that target various rare diseases. Some of their well-known products include Naglazyme, Vimizim, Kuvan, Palynziq, and Brineura, which are approved for the treatment of conditions like MPS VI, MPS IVA, PKU, PKU with hyperphenylalaninemia, and CLN2 disease (a form of Batten disease).
The company's research and development efforts are focused on advancing the science behind genetic medicine and identifying potential therapies for additional rare diseases. BioMarin also collaborates with academic institutions, research organizations, and other biotech and pharmaceutical companies to leverage their expertise and expand their capabilities.
Overall, BioMarin Pharmaceutical Inc. strives to make a meaningful impact in the lives of patients with rare genetic diseases by developing innovative treatments that address the root causes of these conditions, potentially improving health outcomes and quality of life for those affected.