This website is intended only for US healthcare professionals; if you are not a US healthcare professional, please visit www.BioMarin.com
This site offers select publications to support scientific exchange. Content on investigational therapeutics or uses of approved products does not confirm safety or efficacy. BioMarin does not endorse off-label use of its medicines. Clinical study results reflect knowledge at the study's completion and may not be current. Review the methodology, limitations, and authors' financial disclosures for details. This information is not medical advice and should not replace independent medical judgment or further research. Product information may vary by country. Please refer to local product information for indications and safety details.
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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:

Pipeline and Clinical Development Programs

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 the Pipeline and Clinical Development Programs page