BioMarin Pharmaceutical Inc. is a leading global biotechnology company specializing in the development and commercialization of therapies for people with serious and life-threatening rare genetic diseases. Its portfolio includes eight approved treatments targeting conditions such as mucopolysaccharidosis types I, IVA, and VI (branded as Aldurazyme, Vimizim, and Naglazyme), phenylketonuria (Kuvan), achondroplasia (Voxzogo), Batten disease (Brineura), Lambert-Eaton myasthenic syndrome (Firdapse), and hemophilia A (Roctavian). These therapies encompass enzyme replacement therapies, gene therapies, and small molecule drugs designed to address the underlying genetic causes of these disorders. BioMarin Pharmaceutical Inc. operates in more than 70 countries across the United States, Europe, Latin America, the Middle East, and Asia Pacific, serving patient communities through innovative genetic science and molecular biology expertise. Founded in 1997 and headquartered in San Rafael, California, the company maintains a robust pipeline of clinical and preclinical candidates focused on skeletal dysplasias, liver diseases, and muscular dystrophies, reinforcing its pivotal role in the rare disease biotechnology sector.