BioMarin Pharmaceutical Inc.

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

BioMarin employees around the world have come together to celebrate #PrideMonth and demonstrate allyship for the LGBTQIA+ community. From our sites in San Rafael and Novato to Shanbally and São Paolo, members of our PRIDE employee resource group and allies across our company held flag raisings, hosted guest speakers and coordinated other employee activities. We’re looking forward to closing the month on a strong note at the SF Pride Parade this weekend!

Bone growth is regulated by a complex network of biological signaling pathways. To develop medicines for skeletal dysplasias and growth-related conditions, our research over the past two decades has been driven by a fundamental understanding of which pathway to target by leveraging one of the body’s own natural processes – and we’ve validated our approach through extensive preclinical and clinical studies. Learn more about our scientific approach: https://bit.ly/4cvb5lf

Our teams in Ireland have been spending time in their local communities inspiring students to learn about science, technology, engineering and math (STEM). In Shanbally, we recently hosted a cohort of students for a week during their Transition Year – part of the Irish secondary school program designed to act as a bridge between the junior and senior years. The year is meant to help students develop social and work-related skills. More than 30 students experienced firsthand what working at our manufacturing site in Shanbally is like and learned about the different STEM careers available. In Dublin, employees have started an initiative called STEM Heroes focused on creating excitement for STEM among primary school students by making the subject matter more accessible and highlighting real-world applications. A team of BioMarin employees recently visited Realt na Mara National School, providing interactive demonstrations and experiments to open the students’ eyes to the possibilities of STEM.

At #ICCBH2024 we were pleased to share new data from our robust clinical development program in growth-related conditions, including #achondroplasia and #hypochondroplasia. We remain committed to improving the lives of those living with growth-related conditions. Learn more: https://bit.ly/3X9xKiN

C-type natriuretic peptide (CNP) is a signaling protein that plays a key role in bone growth. It works with NPR-B (natriuretic peptide receptor-B) to block FGFR3 signaling and enable bones to grow. At BioMarin, we’re developing treatment options that target the root cause of genetic growth-related conditions, where changes in the FGFR3 gene cause the body to continuously send out signals to slow bone growth. Read more: https://bit.ly/4cvb5lf

Adults with severe #hemophilia A can experience a host of challenges, including frequent infusions and injections. The research we shared at #ISTH2024 reflects our commitment to addressing the needs of adults with #hemophiliaA. Learn more: https://bit.ly/3X9xKiN

Our leadership in developing medicines for children with growth-related conditions is driven by a deep understanding of science. At the core is C-type natriuretic peptide (CNP), a master regulator of bone growth. Across the spectrum of human height, we learned that naturally higher or lower levels of CNP lead to taller or shorter stature, similar to a “dimmer knob” that can control your lighting. While FGFR is also involved in bone growth, its effects are more like an “on-off switch,” which can’t be fine-tuned in the same way. Our research into CNP is evaluating how it can target a particular cellular signal to seek a specific effect while minimizing off-target effects. Learn more about our science-driven approach: https://bit.ly/4cvb5lf