Innovative therapeutics


Patient Advocacy

At BioMarin, we have been focused on treating rare diseases and conditions for more than 20 years, and we could not do it without close collaboration with our patients and patient advocates. We recognize the importance of integrating the patient voice starting at research and development and continuing even after a medicine is approved to best serve the unique needs of each community.


BioMarin Investigational Medicines

At BioMarin, we are committed to the development of new therapies for rare genetic diseases and conditions. Successful clinical trials are required to gain regulatory approval for new medications to advance patient care, as well as to support existing products.

›› What is a clinical trial

Clinical Development Program Resources & Updates

You can view the latest program updates for patient communities listed below:

Our dedicated staff covers multiple regions, attends and hosts important events, provides valuable educational resources and works with a broad range of organizations to make sure that the patient’s voice is integrated into the drug development process and beyond.

Our Therapeutic Areas

BioMarin has several approved products on the market for the below disease areas.

BioMarin in the Community

We have partnered closely with numerous patient advocacy organizations and their members to listen to and understand communities, highlight clinical trial opportunities and develop educational materials to build awareness of rare diseases across the world.

For a list of patient organizations and resources, please click here.

Patient/Advocate Stories

In partnership with Believe Limited and the Foundation for Arts and Healing (FAH), BioMarin sponsored a three-day musical theater and arts workshop for the bleeding disorders community. The musical workshop was directed by hemophilia advocate and Believe Limited CEO, Patrick James Lynch, and was held in November 2018. The workshop culminated in a Broadway-style performance called Hemophilia: The Musical for local bleeding disorder community members, family and friends. Learn more about Hemophilia: The Musical here.

Patient advocacy event

If you are from an advocacy group and have questions regarding Patient Advocacy activities, please contact:

If you are interested in participating in a BioMarin clinical trial and would like to contact someone to learn more, please send a message to

If you are already participating in a BioMarin clinical trial and wish to report a problem with the study drug or side effect(s), please contact your study doctor as soon as possible, and please emails us at or

RareConnections logo

For patient-support services please, visit
For ongoing product support throughout your treatment journey, or if you need help enrolling so you may receive patient assistance, please connect with a Case Manager by email or call 1-866-906-6100.



Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS



Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

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