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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). BioMarin was the first company to provide therapeutics for ...
Show comments. (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 ...
Ultragenyx Pharmaceutical Inc. was founded in 2010 by Emil Kakkis based on his history of developing therapies for rare disease starting at Harbor-UCLA Medical Center, continuing through his role as chief medical officer of BioMarin and as founder of the EveryLife Foundation.
(Reuters) - BioMarin Pharmaceutical said on Tuesday it will price its therapy for severe hemophilia A at 28,933.53 euros ($31,725.62) per vial for insurers covering a majority of the population in ...
February 26, 2024 at 3:50 PM. (Reuters) - BioMarin Pharmaceutical has received a subpoena from the U.S. Department of Justice seeking information about the company's sponsored testing programs ...
Vosoritide. Vosoritide, sold under the brand name Voxzogo, is a medication used for the treatment of achondroplasia, [3] [4] [5] a genetic condition that causes severely short stature and disproportionate growth. [4] Achondroplasia is caused by a genetic mutation that increases the activity of a certain growth regulation gene called fibroblast ...
That said, BioMarin's commercial portfolio is expected to generate sales of more than $1 billion by 2018. Looking at the issue this way, it's plausible that an acquirer could justify a tender ...
KEGG. D12434. Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A. [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 ...