BioMarin Pharmaceuticals Inc
BioMarin's focus is on rare-disease therapies. Genzyme markets Aldurazyme through its joint venture with BioMarin, and BioMarin markets Naglazyme, Vimizim, and Brineura independently. BioMarin also markets Kuvan and Palynziq to treat the rare metabolic disorder PKU. Voxzogo (vosoritide) was approved in achondroplasia in 2021. BioMarin's Roctavian (hemophilia A gene therapy) was approved in Europe in 2022 and in the US in 2023.
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BioMarin's focus is on rare-disease therapies. Genzyme markets Aldurazyme through its joint venture with BioMarin, and BioMarin markets Naglazyme, Vimizim, and Brineura independently. BioMarin also markets Kuvan and Palynziq to treat the rare metabolic disorder PKU. Voxzogo (vosoritide) was approved in achondroplasia in 2021. BioMarin's Roctavian (hemophilia A gene therapy) was approved in Europe in 2022 and in the US in 2023. Show Less
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