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BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States , South America , Asia , and Europe . BioMarin's core business and research is in enzyme replacement therapies (ERTs).
This listing is limited to those independent companies and subsidiaries notable enough to have their own articles in Wikipedia. Both going concerns and defunct firms are included, as well as firms that were part of the pharmaceutical industry at some time in their existence, provided they were engaged in the production of human (as opposed to veterinary) therapeutics.
During his tenure at BioMarin, Kakkis guided the development and approval of two more treatments for rare disorders, MPS VI and PKU and has contributed to the initiation of seven other treatment programs for rare disorders, three of which are now in clinical development. Kakkis is board certified in both Pediatrics and Medical Genetics.
(Reuters) -The U.S. Food and Drug Administration on Thursday approved BioMarin Pharmaceutical's gene therapy for severe hemophilia A, the company said, giving patients with the inherited bleeding ...
(Reuters) - BioMarin Pharmaceutical has received a subpoena from the U.S. Department of Justice seeking information about the company's sponsored testing programs related to two treatments, the ...
Orphan drug specialist BioMarin Pharmaceutical enjoyed a notable week. It started with the U.S. Food and Drug Administration, or FDA, officially approving the company's enzyme replacement therapy ...
Vosoritide was developed by BioMarin Pharmaceutical and got orphan drug status in the US as well as the European Union. [ 5 ] [ 7 ] [ 13 ] [ 9 ] The safety and efficacy of vosoritide in improving growth were evaluated in a year-long, double-blind, placebo-controlled, phase III study in participants five years and older with achondroplasia who ...
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