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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).
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 ...
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.
BioMarin taught big biotech companies a thing or two about making money this year. Here are three lessons large competitors likely learned watching BioMarin succeed in treating rare and ultra-rare ...
(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 ...
(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 ...
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BioMarin stopped providing the drug for free at the beginning of the year after negotiations with Belgian health authorities regarding reimbursement of the product repeatedly failed. This caused the parents to start legal proceedings to force the company to keep providing the medicine free of charge.