BioMarin Pharmaceutical
BioMarin Pharmaceutical is a multinational biotechnology company that specializes in the research, development, and commercialization of innovative pharmaceuticals for rare genetic diseases. The company was founded in 1997 and is headquartered in San Rafael, California.
History
BioMarin Pharmaceutical was established in 1997 by Christopher Starr and Grant Denison. The company's initial focus was on the development of enzyme replacement therapies for genetic diseases that were not being addressed by the larger pharmaceutical companies.
Products
BioMarin Pharmaceutical has a diverse portfolio of products that are used to treat a variety of rare genetic diseases. These include:
- Naglazyme (galsulfase): An enzyme replacement therapy for the treatment of Mucopolysaccharidosis VI (MPS VI).
- Kuvan (sapropterin dihydrochloride): A synthetic form of the enzyme cofactor BH4 used in the treatment of Phenylketonuria (PKU).
- Vimizim (elosulfase alpha): An enzyme replacement therapy for the treatment of Mucopolysaccharidosis IV (MPS IV).
- Brineura (cerliponase alfa): An enzyme replacement therapy for the treatment of Neuronal Ceroid Lipofuscinosis (CLN2), a form of Batten disease.
- Palynziq (pegvaliase): An enzyme substitution therapy for the treatment of adults with PKU.
Research and Development
BioMarin Pharmaceutical is committed to the research and development of new treatments for rare genetic diseases. The company's R&D efforts are focused on enzyme replacement therapies, gene therapies, and other novel approaches to treating these diseases.
See Also
References
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Contributors: Prab R. Tumpati, MD