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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).
By Pratik Jain (Reuters) -The U.S. health regulator on Friday approved the expanded use of BioMarin Pharmaceutical's once-daily injection to treat children under the age of 5 with the most common ...
BioMarin said the subpoena was not related to the safety and efficacy of the therapies. ... BioMarin Pharmaceutical has received a subpoena from the U.S. Department of Justice seeking information ...
By Nathan Gomes (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 ...
BioMarin (BMRN), a leader in the phenylketonuria segment, initiates development of its first gene therapy for treating the rare genetic enzyme deficiency disorder.
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 ...
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 ...
[1] [3] It was developed by BioMarin Pharmaceutical. [4] [5] [6] Valoctocogene roxaparvovec is made of a virus (AAV5) that has been modified to contain the gene for factor VIII, which is lacking in people with hemophilia A. [3] It is an adeno-associated virus vector-based gene therapy. [1] It is given by intravenous infusion. [3]