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

Patient Community Resources & Clinical Program Updates

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

  • Hemophilia A: BMN 270

    • Gene therapy is being investigated for a range of conditions to evaluate its safety and efficacy. BioMarin has created an informational resource discussing the science behind the ongoing research in gene therapy.
      Please visit to learn more.

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.

  • PKU
    • If you’re interested in patient stories, resources and community events related to PKU, please visit .
    • Live Unlimited PKU is raising awareness of PKU, and the challenges which adults living with the disease face on a day-to-day basis. Join our community on Facebook.
  • Morquio A (MPS IVA)
    • To learn more about Morquio A and find educational resources, please visit
  • CLN2 Disease
    • Families affected by CLN2 disease can connect with a growing community of support and advocacy groups at
  • MPS VI
  • MPS I

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.


Overview of Achondroplasia and the BioMarin Clinical Trial Program

Overview of Achondroplasia and the BioMarin Clinical Trial Program

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.

Book cover: 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.

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

Awareness Dates:

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 email 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



Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

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