Biomarin Pharmaceutical
Biomarin Pharmaceutical
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moreBioMarin Pharmaceutical Inc. was established in 1997 and has since positioned itself as a notable player in the biotechnology and pharmaceutical sectors. Specializing in innovative therapies for rare genetic disorders, the company is headquartered in San Rafael, California, United States. It focuses on enzyme replacement therapies among other drug developments tailored to particular medical needs. With a portfolio of key pharmaceutical products, BioMarin has developed a series of prominent brands vital for treating specific conditions. Vimizim is designed for patients with Morquio A syndrome, while Naglazyme addresses MPS VI. Both Kuvan and Palynziq are treatments for phenylketonuria, and Brineura is crafted for the treatment of CLN2 disease, a form of Batten disease. In collaboration with Sanofi Genzyme, they also market Aldurazyme for patients with MPS I. Due to the rarity of the diseases they target, BioMarin has an expansive market presence worldwide, encompassing the United States, European nations, Latin America, the Middle East, Asia, and the Pacific. The company's reach extends across many different countries, ensuring those with rare genetic diseases have access to their specialized treatments, regardless of the varied regulatory landscapes they operate within. (Powered by AI)
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Neuronal ceroid lipofuscinosis 2 (CLN2 or Batten disease) is a rare progressive nervous system disease that affects kids between ages 2-4; general symptoms are speech delay, seizures, loss of muscle coordination, muscle twitches, loss of vision, d...