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For 25 years, BioMarin has been dedicated to transforming lives through genetic discovery. We develop targeted therapies that address the root cause of the genetic conditions that it seeks to treat, helping to better the lives of those suffering from rare genetic disease – all through 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.

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.

Investigational Medicines

At BioMarin, we are committed to the development of new therapies for 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 ›

Phenylketonuria (PKU) Clinical Trial Information

PEGASUS Study & Logo

BioMarin is conducting a global clinical trial for adolescents with phenylketonuria (PKU) aged 12 to 17 years. Learn about the PEGASUS study to help advance PKU research.

Learn More

Patient Community Resources & Clinical Program Updates

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

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.


image: overview of achondroplasia

Listen as our expert faculty discuss achondroplasia, the most common form of dwarfism, and share an overview of the clinical program for BMN 111, the investigational treatment BioMarin is studying in children with achondroplasia. BMN 111 is being researched, it has not been approved for use or been determined to be safe or effective. This is a recording made from a live, educational webinar, hosted by The MAGIC Foundation and sponsored by BioMarin. The webinar is presented by Teresa Tucker, Co-Founder and Patient Advocate for The MAGIC Foundation, and Lynda Polgreen, MD, MS, of The Lundquist Institute at Harbor-UCLA Medical Center.

The video can be watched here.

Image: Children's Book - Albert is like me!

One trial support initiative is a children’s book entitled, Albert Is Like Me!. The Albert book was developed to help the child and caregiver manage long clinic visits as well as study requirements at home. Albert becomes a familiar and relatable character that guides the youngest participants on their clinical trial journey. Once approved by institutional review boards and ethics committees, the book was available to be provided to parents and their young children participating in the BMN 111-206 study for achondroplasia.

An example of the book in US English can be downloaded here.

Awareness Dates


For more information please contact BioMarin Medical Information: medinfo@bmrn.com

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 email us at drugsafety@bmrn.com or productcomplaint@bmrn.com.

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