BioMarin Pharmaceutical Inc.

“The first day we brought her home, we knew we were going to treat her just like any other kid… Our hope for her future is that she conquers the world and does whatever she wants.” With her family’s support and encouragement, KK brings excitement, passion and fearlessness to everything she does, whether it’s soccer, swimming, cheerleading or horseback riding. As we recognize the start of #DwarfismAwarenessMonth, we’re excited to amplify the voices and experiences of people like KK who are living with #achondroplasia and other genetic skeletal conditions. Learn more about her story: https://bit.ly/4e5Nrwz

We're pleased to join our partners at CAMP4 Therapeutics in announcing a new research collaboration aimed at advancing novel therapeutics that increase protein levels by targeting regulatory RNA (regRNA) sequences, which are key elements controlling gene expression. Learn more: https://bit.ly/3ZLa5q0

“Our lives could have been different without that early diagnosis… I’m hopeful that newborn screening continues to expand to cover more conditions and the goals of newborn screening will be fully realized.” Denise Queally still vividly remembers the moment in 2001 when she learned her newborn daughter, Caroline, had screened positive for phenylketonuria (PKU), a rare, genetic metabolic condition. That phone call and the interactions that followed set her family on a path to help Caroline effectively manage her condition and connect with a close-knit community that inspired Denise to become an advocate for people living with PKU and other metabolic conditions. Learn more about Denise’s story, her work and what newborn screening has meant to her family: https://bit.ly/4eJGUrB #NewbornScreening #NewbornScreeningAwarenessMonth

We're excited to share new data at #ASBMR2024 that reinforce our pioneering research into leveraging CNP, one of the body’s own natural signals, and targeting the NPR2 pathway to impact growth in people with skeletal conditions. We’re committed to working to improve outcomes through our continued research. Learn more: https://bit.ly/4cvb5lf

As we’ve gotten to know our 2024-25 RARE Scholars award recipients, we’ve been inspired by their stories and energized by their ambitions. Learn more about this year’s scholars – Allison Lowrie, Allyson Chan, Brian DuVal, Jiayi (Bryan) Yan and William McClintock – and their academic journeys: https://bit.ly/3MX5TMi #BioMarinRAREscholars

“Kainen’s situation changed everything. It just gave you a broader perspective on how things could literally change at the blinking of an eye.” When Kainen received a diagnosis of Duchenne muscular dystrophy, his parents, Marcus and Cathy, recall how it affected their daily lives and outlook on the future. We’re sharing Kainen’s story during #MuscularDystrophyAwarenessMonth to highlight the unmet need that remains for this community, and the importance of continued research and innovation in this space. Learn more: https://bit.ly/4dSw1Un #30DaysOfStrength

Last week at #ISDS2024, we shared new data from CANOPY, our clinical program investigating the potential of CNP in a number of genetic skeletal conditions. Learn more: https://bit.ly/3XwlO9f

“We look at Chris and we see how positive he is about this whole journey of having Morquio A. It’s given us a lot of strength and hope.” Vanessa says her family’s outlook on life changed when her son, Chris, was diagnosed with mucopolysaccharidosis IVA (MPS IVA, or Morquio A syndrome), a rare, progressive condition that can impact organs, bones and other parts of the body. Learn more about Chris’s story in the next installment of our “Patients Are Counting On Us” video series: https://bit.ly/4eynCFB

Jewell Martin, MA, MBA, PMP, RAC, Director of R&D and Regulatory Policy at BioMarin, moderated a panel discussion on lessons learned in clinical trial diversity at this week’s Regulatory Affairs Professionals Society (RAPS) Convergence in Long Beach, California. Panelists from both the public and private sectors discussed the real-world application of the FDA’s Diversity Action Plan draft guidance, noted early successes and challenges, and highlighted initiatives to raise awareness of clinical trials among historically underrepresented communities. Jewell is a member of BioMarin’s Clinical Trial Diversity Working Group, a cross-functional team of leaders across our company who play a role in designing and conducting clinical trials. Learn more about the focus areas our working group identified: https://bit.ly/3B8XliO

This Hispanic Heritage Month, we’re grateful for and proud to recognize the efforts of our Latinx employee resource group (ERG). Since its founding more than three years ago, the Latinx team has been instrumental in helping enable leadership and mentorship opportunities for employees. The group has also empowered local students to pursue careers in STEM by serving as role models and raising awareness about professional opportunities. The Hispanic/Latino Professional Association recognized the impact of the ERG’s work this year, naming BioMarin to its list of Best Places to Work for Hispanics/Latinos.