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BioMarin's core business and research is in enzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I), by manufacturing laronidase (Aldurazyme, commercialized by Genzyme Corporation). BioMarin was also the first company to provide therapeutics for phenylketonuria . [5] [6]
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 ...
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.
Lon Ray Cardon FMedSci is an American human geneticist who is President and Chief Executive Officer of The Jackson Laboratory.Previous to joining The Jackson Laboratory in 2021, he had roles as Chief Scientific Officer and Chief Scientific Strategy Officer at BioMarin Pharmaceutical and senior vice president at GlaxoSmithKline, where he worked to translate the results of genetic research ...
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Elosulfase alfa is used in enzyme replacement therapy; a 2014 study confirmed it was effective on young patients with Morquio syndrome type A. [4] Treatment with this medication was most effective upon respiratory symptoms, activities of daily living and growth, as confirmed in a 2015 paper. [5]
[9] [10] In 2012, it in-licensed patents covering the use of amifampridine phosphate to treat LEMS for the North American market from BioMarin. [11] In 2012, while BioMarin had a Phase III trial ongoing in the US, it licensed the US rights to 3,4-DAPP, including the orphan designation and the ongoing trial, to Catalyst Pharmaceuticals. [12]