BioMarin Pharmaceutical Inc. (United States)

BioMarin Pharmaceutical is a global biotechnology company developing therapies for rare genetic diseases. Based in San Rafael, California, BioMarin creates treatments for phenylketonuria (PKU), mucopolysaccharidosis (MPS), Batten disease, and achondroplasia. Key products include Vimizim, Naglazyme, and Aldurazyme for MPS disorders, Palynziq for PKU, and Voxzogo for achondroplasia (dwarfism). BioMarin’s enzyme replacement and gene therapies transform lives of patients with rare inherited conditions.