This website is intended only for US healthcare professionals; if you are not a US healthcare professional, please visit www.BioMarin.com
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BioMarin Medical

An online medical resource for healthcare providers in the United States to learn about our latest research, ongoing clinical trials, and the therapeutic areas where we focus

Disease States and Programs

BioMarin has therapies either in development or approved by the US Food and Drug Administration (FDA) in the following disease areas:

Our Pipeline

At BioMarin, we push the boundaries of medical possibility to make a transformative impact. We are dedicated to improving the lives of individuals living with rare genetic disorders. We do so by investing in groundbreaking research and developing innovative treatments.

Learn more about BioMarin research and clinical trials with treatments that utilize the following therapeutic technologies:

Gene Therapies

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Small Molecules

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Antisense Oligonucleotides

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Biologics

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Explore our pipeline