BioMarin is a global biopharmaceutical company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. BioMarin remains steadfast to its original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. We are world leaders in metabolic disease innovation with multiple products commercialized and a growing pipeline of product candidates to address unmet medical needs. We continue to fuel our R&D engine by looking for opportunities that align with our strengths and competencies. And we relentlessly pursue exciting, early-stage science that has the potential to change the course of disease.