BioMarin Pharmaceutical Inc.

The FDA has approved expanding the use of our treatment to now include children of all ages with CLN2, a form of Batten disease, regardless of whether they show symptoms. Learn more: https://bit.ly/4bZfg8c

As the 2024 National PKU Alliance Conference comes to a close, we are inspired by the stories we heard and remain committed to advancing our research and support for the #PKU community. #NPKUA2024Conference

We were honored to welcome the latest cohort of BioMarin fellows on a warm, sunny day at our San Rafael campus. BioMarin now offers two postdoctoral fellowship programs: The Regulatory Affairs Fellowship (in partnership with Howard University) and the Research and Early Development Fellowship. Our two-year programs are aimed at providing early-career researchers with the opportunity to grow their experience at BioMarin and gain a deeper understanding of the biopharmaceutical industry. A special thank you to Dr. Earl Ettienne, assistant dean of Graduate Programs and Industrial Partnerships at Howard University, for joining us to welcome the fellows and for his ongoing support of the program.

As the summer continues, so does our connection with the #PKU community. Did you know that there are family camps across the U.S. that bring the #phenylketonuria community together? These camps enhance support networks and connect families. Learn more here: https://bit.ly/3Koh4MO

Meet PAH: a gene that plays a key role in breaking down an amino acid called phenylalanine found in many foods. Changes in the PAH gene can impact the body’s ability to metabolize phenylalanine, affecting brain development and function. For many years, we have worked to develop treatments that can make a positive impact for people living with phenylketonuria (PKU), which is caused by alterations in the PAH gene.

From childhood, those diagnosed with #PKU tend to feel different than other children. As Jonathan grew older, went to school and got involved in playing sports, he searched for ways to hide his dietary restrictions and other differences from his peers. Learn more about Jonathan’s story here: https://bit.ly/3W06sdD

We’re looking forward to connecting with the #PKU community later this week at the National PKU Alliance 2024 National PKU Alliance Conference, where we’re sponsoring several educational opportunities for teens and adults living with PKU. Learn more: https://bit.ly/3XYgs8z. #NPKUA2024Conference

Chondrocytes are cells in the cartilage that line up to form new bone. Think of these as architects in your body, sending out and receiving signals that control the process of bone growth. One signal that tells chondrocytes to slow down is a gene called FGFR3. At BioMarin, we’ve been working for nearly two decades to understand how changes in the FGFR3 gene can affect people with growth-related conditions, including achondroplasia.

No matter how someone grows, we all “rise with love.” That’s the underlying message behind Rising Love – a children’s book that uses the analogy of baking bread to describe the experience of growing up with #achondroplasia. Just as bread dough might rise to different shapes, sizes and textures, bones grow uniquely in children with achondroplasia, the most common form of dwarfism. We regularly host community events to read Rising Love and bake with children living with achondroplasia and their families, including a recent one hosted with The Chandler Project in Baltimore. Participants worked together to bake focaccia with local chef Ryan Scalfari. Learn more about local baking events across the U.S. and download the free book here: https://lnkd.in/gZgU-AMe #RisingLove

“I hope there continues to be new options for people and that, more and more, the limitations of living with PKU can become a thing of the past.” Today as we recognize International PKU Day, we’re proud to continue to help people living with #PKU like Jonathan navigate their journeys with this rare genetic condition. We recently spent some time getting to know Jonathan, learning about his experience with PKU and his hopes for the future. Read more: https://bit.ly/3W06sdD #PKUDay2024