BioMarin Pharmaceutical Inc.

02/28/2026

Ahead of , we spoke with two members of the BioMarin Leadership Team – Chief Business Officer James Sabry and Chief R&D Officer Greg Friberg – and asked them to reflect on their time in medical school. They recall a common adage taught to young doctors – one that unintentionally resulted in clinicians overlooking uncommon or rare conditions, often referred to as “zebras.” Over time, this led to zebras becoming a powerful symbol for the rare disease community in the United States.

As we recognize Rare Disease Day today, we’re proud to show our stripes as a company that listens closely and looks for zebras when we hear hoofbeats.

02/28/2026

We were inspired by the incredible energy our teams brought to this year, as we joined patient communities around the world in raising awareness about the more than 10,000 rare diseases impacting an estimated 300 million people globally. Our employees got into the spirit by showing their stripes in zebra print, sharing their Rare Disease Day colors, pledging support on social media and coming together with a unified sense of purpose.

We were especially grateful for the opportunity to spend time earlier this week with Allyson Chan, a young adult living with mucopolysaccharidosis (MPS) type IVA (also known as Morquio A syndrome). Allyson and her mother, Christina, joined us for a special all-hands meeting at our San Rafael headquarters that was broadcast to our employees around the world. In conversation with our Letticia Atkins, Allyson shared her personal journey, including what it was like growing up with MPS IVA and how she has successfully navigated the transition to college. Now a sophomore in college in Los Angeles, Allyson is a two-time recipient of our RARE Scholars award, which provides financial support to students with rare genetic conditions who demonstrate exceptional leadership and engagement. She is pursuing a degree in politics and writing for the school’s student-run newspaper. Allyson also toured our labs and connected with several of our team members who have helped research and develop medicines for people living with rare genetic conditions like her.

These moments remind us why our work matters and deepen our connection to the communities we serve. Thank you to Allyson and Christina for graciously sharing their lived experiences with us, and to our teams around the world for your enthusiasm in helping us recognize Rare Disease Day 2026.

02/27/2026

We’re excited to announce that we are now accepting applications for our annual RARE Scholars program for the 2026-27 academic year!

Since 2018, RARE Scholars has provided financial support to U.S. college students living with rare conditions, including , , ( ) and ( ), who have demonstrated exceptional leadership and engagement in school and community activities.

This year, we’re proud to recognize the critical role of rare disease caregivers by expanding our program eligibility criteria to include legal guardians of individuals living with one of these conditions. The program will continue to include five slots for people living with achondroplasia, Batten disease, MPS or PKU, with an additional slot held for an eligible legal guardian.

Applications will be accepted through March 30. Apply today: http://ms.spr.ly/6180Qj6r4

02/26/2026

Over the past several months, we’ve had the privilege to partner with the National MPS Society on the campaign, highlighting the resilience, individuality and lived experiences that make the mucopolysaccharidosis (MPS) and mucolipidosis (ML) community so extraordinary. Each story shared has been a powerful reminder that representation matters, and that behind every diagnosis is a person, a family and a journey that deserves to be seen and understood.

We’re deeply grateful to the individuals and families who helped bring Faces of MPS to life, including Kelley, Kashton, Juliet, Dana, Lilah, Jennifer, Jacob, Savannah, Daili, Leidy, Makenzie and their families. Thank you all for sharing your journeys. We look forward to continuing to help raise awareness and amplify stories from the MPS and ML community.

Learn more about MPS and ML on the National MPS Society website: http://ms.spr.ly/6189Qbr9L

02/26/2026

BioMarin was in Sacramento at the California State Capitol celebrating Rare Disease Week alongside patient advocates, researchers and policymakers as the California Legislature introduced a statewide resolution recognizing . We were pleased to see several members of the communities we serve – including the , and ( ) communities – recognized by the California Legislature in the resolution.

Several individuals we have partnered with were honored for their advocacy efforts on the assembly floor, including Patricia Espinal, who lives with MPS VI, and Suzette James, who has two children living with a form of Batten disease called CLN2. We’d also like to thank Dr. Pedro Sanchez for sharing his story as both a father of a child with a rare condition and a physician who cares for people living with rare diseases.

California’s Rare Disease Caucus hosted a press conference, during which Bridget Yates, a BioMarin scientist in Research and Early Development, shared remarks about working tirelessly to serve patients with significant unmet needs. Bridget has spent more than 20 years studying rare genetic diseases, with a focus on discovering and developing life-changing medicines.

This resolution and the accompanying press conference are significant milestones, increasing visibility for rare conditions and supporting ongoing advocacy efforts to expand newborn screening and access to treatment for patients. We're encouraged to see patient communities being given greater recognition and a platform to contribute to policy advocacy goals in our home state. Thank you to California Life Sciences - CLS and to Assemblymember Rick Zbur, Assemblymember Diane Dixon and all legislators in the Rare Disease Caucus.

02/25/2026

“The shared decision-making process is critical to having the best outcomes for patients. It really starts with listening and meeting families where they’re at.”

We were proud to once again sponsor a panel at this week’s Rare Disease Summit, hosted by Boston Globe Events. Titled “Stronger Together: Navigating Shared Decision-Making in Genetic Skeletal Conditions,” the panel explored the experiences and perspectives of people living with skeletal dysplasias. Kristen DeAndrade, Founder and President of The Little Legs Big Heart Foundation, moderated an empowering discussion with Dr. Jennifer Arnold, Executive Director, Immersive Design Systems; Cristian Garcia from Thee Garcia Family, a social media influencer, entrepreneur and father of two children with ; and Becky Curran, a TEDx Motivational Speaker and Global Disability Inclusion Advocate, Becky Motivates LLC.

The conversation centered on lived experience in care decisions, navigating complexities faced by individuals, parents and caregivers, and advancing shared decision-making toward becoming the standard for the best holistic health outcomes.

A heartfelt thank you to all the panelists for sharing their time and wisdom, and to The Boston Globe for partnering with us to create this important conversation.

02/24/2026

Today, we join the Noonan Syndrome Foundation in recognizing Noonan Syndrome Awareness Day, recognized every Feb. 23 during Noonan Syndrome Awareness Month. We're committed to learning more about the lived experiences of this community as we work to help expand options for people living with this rare genetic skeletal condition. Learn more at www.teamnoonan.org.

🧬 February is Noonan Syndrome Awareness Month!

Each year during the month of February, the Noonan Syndrome Foundation (NSF) shares one independent fact per day to help educate, empower, and unite our community. We encourage everyone—families, advocates, friends, and supporters—to save & share these daily facts across your social media platforms to help raise awareness, strengthen advocacy efforts, and spread understanding.

Every fact shared throughout the month has been reviewed and approved by the NSF Medical Advisory Board, in conjunction with the NSF Board of Directors, to ensure the information is accurate, evidence-based, and current.

Whether you’re new to the Noonan Syndrome community or have been walking this journey with us for years, we hope these daily facts bring deeper insight, meaningful learning, and a greater understanding of Noonan syndrome.

❤️💙Our goal is simple: provide trustworthy information and inspire broad sharing—because awareness creates action, and action creates change.

And always remember…
🌼 We were all BORN TO BLOOM! 🌼

02/20/2026

Team Daly Helixes narrowly edged Team Genetic Trailblazers in a spirited seesaw battle yesterday during BioMarin's Black History Month Jeopardy-style Game Show, hosted by our Black Employee Resource Group (BERG).

In front of a standing-room-only crowd at our San Rafael headquarters, the match successfully brought to life several of BioMarin's operating principles, including “Have fun” and “This is a competition.” The two teams matched wits in categories that included Black Politics and Law, Black Trailblazers and The Mother Land, among others, paying homage to the 100th Black History Month and educating employees along the way. Trivia answers highlighted important moments in history, as well as Black leaders and pioneers in industry, science, politics and art.

With both teams locked in a dead heat after perfect performances during regulation, the Daly Helixes – named after Marie Maynard Daly, the first Black woman to earn a Ph.D. in chemistry – pulled out a win in overtime. Bekalu (BK) Abebe, who joined Greg Friberg, Marni Kottle and Shola Fafunso to make up the Daly Helixes, said the team was thrilled to earn the victory against tough competition.

“This was a great team win,” said BK, an engineer in the company’s Manufacturing organization. “It was important for us to honor the stories that continue to shape our world. And of course, a little friendly competition is part of how we live our BioMarin operating principles.”

The Genetic Trailblazers, starring Amy Wireman, Brian Mueller, Courtney Banks and Jason Lewis, were pleased to help engage employees around Black history and took the narrow loss in stride.

“I had a blast competing with my teammates,” said Courtney, who works in our Finance department. “Great job to all who participated and a big thank you to our BERG leaders for putting together such a fun and engaging event."

02/18/2026

For Chieze Ibeneche-Nnewihe, formative childhood experiences included conducting chemistry experiments on the porch with her father and two sisters, collaborating on circuit board projects, and engaging in dinner conversations about scientific phenomena such as condensation forming on cold glasses.

Raised between the Netherlands and Nigeria by parents who were an engineer and a broadcast journalist, Chieze developed a strong sense of curiosity from an early age. Her upbringing in a family dedicated to inquiry and discovery significantly influenced her worldview and guided her eventual decision to pursue a career in healthcare.

Read more about Chieze’s evolution from front-porch scientist into a leading role on BioMarin’s Business Development team, helping shape the company’s pipeline, portfolio and impact for patients: http://ms.spr.ly/6182QViqe

02/13/2026

“Black History Month helps contextualize where we are, where we’ve been and where we’re headed. We can live in both truths – honoring progress while acknowledging the work still ahead.”

This was among the sentiments shared by our Black Employee Resource Group members as we asked them to reflect on their experiences within the organization and to share the significance of acknowledging moments like Black History Month as an essential element of fostering a culture of inclusion at BioMarin. Our colleagues Asha Dampier, Alexandre Eugene, Nina Orike and Remi Murphy shared their perspectives as we continue commemorating Black History Month throughout February.

02/12/2026

Makenzie is curious and thoughtful, with a strong sense of self‑advocacy. Her three dogs, Harley, Mimi and Capone, are constant companions and a source of comfort. She is an amateur photographer and proud foodie who enjoys traveling to try new restaurants and different coffee shops.

She shares a close bond with her mom, Andrea, as well as her two younger siblings: Taylor, who like Makenzie is living with (MPS) type VI, and Kaleigh, their youngest sister.

Makenzie was diagnosed with MPS VI, a progressive, rare genetic condition also known as Maroteaux‑Lamy syndrome that leads to the buildup of complex carbohydrates, causing complications to multiple organs, at the age of 4.

At the time, Andrea had not noticed anything unusual until a routine checkup raised concerns. Genetic testing later confirmed the diagnosis for both Makenzie and Taylor, who were among the first patients with MPS VI their pediatrician had met.

Over the years, Makenzie has faced significant medical challenges, including spinal decompression and fusion surgeries in her neck and back, as well as airway interventions, including a tracheostomy.

Throughout this journey, the family says the National MPS Society has been a constant source of connection and support. Andrea has built relationships with many families through social media, and their family has attended numerous MPS conferences over the years, finding community and shared understanding along the way.

As a result, Makenzie, Taylor and Andrea have become people others turn to, especially families navigating the transition from pediatric to adult care. When asked what advice they would give to families newly diagnosed with MPS, Andrea doesn’t hesitate.

“Don’t blame yourself.” she says. “If possible, involve your child in their medical care early and often. Every decision Makenzie has faced has been discussed together as a family.”

Makenzie echoes the importance of that sense of empowerment, encouraging others to find their voices and use them.

“Speak up if people aren’t listening,” Makenzie advises. “Don’t let anyone walk over you and be your own advocate.”

Looking to the future, Makenzie hopes for continued consistency in her care so she can remain healthy and stable. She and her family are eager to make new memories through travel, including attending this year’s National MPS Society Family and Scientific Conference in Cincinnati, with hopes of one day visiting New York together.

Read more about Makenzie’s story on the National MPS Society website: http://ms.spr.ly/6188QRpBQ

02/09/2026

When Christine O’Brien’s children were diagnosed with generalized arterial calcification of infancy (GACI), caused by ENPP1 deficiency, there was no roadmap to guide their journey and no real support system to look to for answers. Information was limited, and Christine and her husband Michael left the hospital carrying fear, uncertainty and a growing list of unanswered questions.

“We left the NICU with this little yellow piece of paper, which had the name and phone number of the only other family in the Boston area who faced this diagnosis,” she recalls. “And so It was this beacon of hope that there was somebody out there that was dealing with this.”

Searching for connection, Christine started her advocacy journey by reaching out to others facing a GACI diagnosis, one family at a time. Those early conversations became relationships, and over time, those relationships grew into something bigger: GACI Global, a patient advocacy organization focused on connecting families affected by GACI or related conditions to each other and the medical community.

As we continue working to advance scientific progress for people living with ENPP1 deficiency, we’re looking forward to connecting with the global community at the GACI Global Patient and Family Conference this weekend in Orlando.

Learn more about GACI Global: http://ms.spr.ly/6184QPGka