BioMarin (San Rafael, CA). 2024 revenue ~$2.5B. Rare disease leader. Voxzogo (vosoritide, achondroplasia). Roctavian (valoctocogene roxaparvovec, hemophilia A gene therapy). Naglazyme, Vimizim, Brineura.
12:54:08 AM · Jul 9, 2026
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