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.
Hemophilia Clinical Trial Information
Learn about the BioMarin gene therapy clinical trial program for BMN 270, an investigational gene therapy for hemophilia A. Take a closer look at how clinical trials for hemophilia A are being designed and the current progress of BMN 270. This webinar recording also reviews the Complete Response Letter (CRL) from the FDA and what
Patient Community Resources & Clinical Program Updates
You can view the latest program updates for patient communities listed below.
- Hemophilia A: BMN 270 program update page⌃
- Achondroplasia: BMN 111 program update page⌃
- Phenylketonuria: BMN 307 program update page⌃
- Hereditary Angioedema: BMN 331 program update page⌃
- Duchenne Muscular Dystrophy: BMN 351 program update page⌃
- Alpha-1 Antitrypsin Deficiency: BMN 349 program update page⌃
- Primary Hyperoxaluria Type 1: BMN 255 program update page⌃
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.
Collaborations
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.
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
- Rare Disease Day –
- Bleeding Disorders Awareness Month –
- World Hemophilia Day –
- National PKU Awareness Month –
- MPS Awareness Day –
- HAE Day –
- International Batten Awareness Day –
- International PKU Day –
- National Dwarfism Awareness Month –
- National PKU Awareness Day –
- International Day of Persons with Disabilities –
Contact
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.
