We seek the big effect
Strategically focusing on rare diseases with defined genetic defects, BioMarin strives to develop innovative therapies that are first- or best-in-class.
At BioMarin, we view ourselves as a team of pioneers, willing to take risks in the quest to find new solutions to address serious, debilitating challenges in rare diseases, and motivated by the desire to help improve the lives of patients with unmet medical needs.
BioMarin is committed to translating genetic discoveries into transformative medicines. Our approach focuses on diseases with precisely understood mechanisms that are rooted in GENETICS, developing TARGETED therapeutic interventions that address the underlying causes of diseases, utilising the readily ACCESSIBLE endpoints that are proximal to the disease pathway, and enabling TRANSFORMATIONAL changes to the way patients feel, function, and survive.
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Strategically focusing on rare diseases with defined genetic defects, BioMarin strives to develop innovative therapies that are first- or best-in-class.