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Transforming Lives Through Genetic Discovery

Over two decades ago when we first opened our doors, we focused on giving much-needed attention to the underserved communities of those with rare diseases. These rare disease communities mostly affected children and were often ignored. At the time, BioMarin developed the only treatments for these life-altering conditions, giving hope to patients and families.

Throughout our history, we’ve worked tirelessly to make a difference by pursuing bold science while respecting, educating, and connecting with patients. Through our expertise in genetics and molecular biology, we have been able to develop targeted therapies that address the root cause of the exact conditions we seek to treat.

Our discoveries have led us to countless breakthroughs, best-in-class treatments and many ‘firsts’ in the category. We are grateful to able to better the lives of those struggling with genetic diseases.

As we look forward, we strive to make an even greater impact by applying the same science-driven, patient-forward approach to a broader range of genetic disorders.

The more innovative solutions we develop, the more lives we can impact.

This isn’t just our passion, it’s our purpose.

Image: Son on fathers shoulders
Our History

More than twenty years of transforming lives through genetic discovery.

Learn More about our history
Our Pipeline

We maximize the impact of genetic innovation to develop transformative therapies.

Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.

Learn More Our Pipeline