This website is intended only for United States healthcare professionals; if you are not a United States healthcare professional, please visit www.BioMarin.com
This website was developed and funded by BioMarin and is intended for healthcare professionals only. It offers a non-exhaustive selection of materials, including but not limited to publications, presentations, posters and educational materials, to support scientific exchange.
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.
The information on this website is not medical advice and should not replace independent medical judgment or further research. 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.
As product information may vary by country, please refer to your local health authority regulations and respective approved product information for indications and safety details.
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
BioMarin has therapies either in development or approved by the US Food and Drug Administration (FDA) in the following disease areas:
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
Small Molecules
Antisense Oligonucleotides
Biologics
