BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical develops medicines for rare genetic and metabolic disorders that are often life-threatening. Their approved drugs include treatments for conditions like achondroplasia (a form of dwarfism), mucopolysaccharidosis (a disorder where the body cannot break down certain substances), phenylketonuria (an inherited metabolic disease), and Batten disease. These medicines are typically injections or oral drugs that patients take regularly. The company also has drugs in development for muscular dystrophy and other rare growth disorders.