Rare opportunities, personal impact
At BioMarin, we focus on developing first-in-class, best-in-class therapies that provide significant advances for patients living with severe and life-threatening rare genetic diseases. BioMarin remains steadfast in its original mission – to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mainly children, suffering from diseases so rare that the entire patient population can reach only 1,000 people worldwide. These conditions are usually hereditary, difficult to diagnose, progressively debilitating, have few or no treatment options, and are generally ignored.