Introducing BioMarin Hemophilia—inspired by the aspirations of the hemophilia community, driven to deliver on the ingenuity of gene therapy research. FIND OUT MORE >

"My disability gives me my drive." Mitch does not let living with MPS VI stop him from pursuing his many passions. Hear Mitch’s story >

Tyler doesn't let MPS VI interfere with his daily life. "I try to live as normal a life as I can and do the things that people my age like to do." See how Tyler is managing MPS VI so he can focus on achieving his dreams. Read TYLER's STORY >

”Everyone has struggles and troubles in their life,” she says. “it’s how we deal with them that matters...“ Read KENDRA'S STORY >

“We are literally in a race against time to get ahead of the damage caused by this disease, and as parents, we cannot watch this happen without a fight.” Read AnnaBelle's Story >

Liam used to struggle to keep his Phe levels under control. But now he is better able to manage his PKU. Today he has less to worry about so he can just focus on being a kid. Read Liam's Story >

Watch the 20 year journey of the people of BioMarin finding tomorrows by staying focused on the science to make a big difference for small patient populations. View film >

“What they are doing at BioMarin is changing the lives of families around the world. They are not producing a product. They are truly finding tomorrows.” Learn more >

BioMarin is proud to be named 4th Best Midsize Employer in America by Forbes and 1st Amongst Industry Peers. Learn more >

Watch “The Pursuit of PKU,” which details the history of the rare condition, as well as BioMarin’s 15-year commitment to advancing the standard of care for this community. View film>

The award recognizes Dennis for his contribution in inventing an innovative treatment for breast cancer. Learn More >

Japanese Regulatory Authority, MHLW Approved Brineura (cerliponase alfa) for the Treatment of CLN2 Disease, Ultra-Rare Pediatric Neurodegenerative Disorder in Children Learn More >