This website was developed and funded by BioMarin. This website is intended for healthcare professionals only. Please refer to your local health authority regulations and respective approved product information.

This website was developed and funded by BioMarin. It is intended for EU healthcare professionals; if you are not a EU healthcare professional, please visit

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BioMarin Medical

An online medical resource for healthcare providers 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 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


Small Molecules


Antisense Oligonucleotides



Explore our pipeline