BioMarin Pharmaceutical Inc.

Today we announced a strategic collaboration and global exclusive license agreement with nonprofit organization n-Lorem Foundation to develop an antisense oligonucleotide medicine for people living with ReNU syndrome, a serious and rare neurodevelopmental condition that is caused by variants in the RNU4-2 gene and currently has no approved medicines. ReNU syndrome was identified as a distinct condition in 2024 by an international team of geneticists, following the work of families, advocates and other researchers to raise awareness and accelerate understanding of the genetic variant. As we continue to build out our pipeline, we are excited to partner with the antisense experts at n-Lorem to leverage our leadership in genetic medicines to develop this potential first-in-disease treatment for ReNU syndrome. Read more in our press release: http://ms.spr.ly/6044vCgaI

We're proud to once again support the Massachusetts biotechnology community by sponsoring a LabCentral Golden Ticket, which will be awarded to one promising startup developing innovative therapies or technologies to help transform the lives of people with genetically defined conditions. The winning startup will receive a $50,000 credit toward a year of free bench space for one scientist, plus access to LabCentral's shared infrastructure, services and community. LabCentral serves as an incubator and launchpad for high-potential startups in life sciences and biotech and has provided more than 150 Golden Tickets since the program began in 2013. Applications will be accepted through Friday, Sept. 4, with finalists announced in early October. Apply today: http://ms.spr.ly/6046v71v6

Megan Bull’s path to BioMarin began with a childhood friendship that gave her an early, personal connection to the rare disease community. “I saw the incredible impact physicians and care teams could have,” she recalls, “but I also dreamed of contributing to solutions that could reach and help more people.” Today, as a researcher in Translational Sciences, Megan helps generate reliable scientific evidence that supports the development of therapies for patients. Her story is also shaped by resilience, advocacy and the connections with colleagues that helped her navigate intense, life-changing experiences after joining BioMarin. Read more about Megan’s journey, the role connection has played in her career, and how she brings both rigorous science and human compassion to her work: http://ms.spr.ly/6047vAsdd

Iván is curious and constantly learning, with a passion for music and technology. At age 39, Iván lives in Argentina and works as a digital instructor, teaching older adults how to use technology and build confidence in an increasingly digital world. When he's not leading workshops, he's studying UX/UI design, playing guitar and piano or exploring new software and creative tools. “I hope that what defines me is being someone who is always curious and who enjoys helping others,” says Iván. After years of unanswered medical questions, Iván was diagnosed at 6 years old with #mucopolysaccharidosis (MPS) type 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. What followed was a journey filled with appointments, tests and treatment, shaping both his perspective on life and his resilience. Growing up, there were moments when MPS made him feel different. He was often unable to participate in physical education classes and sometimes felt left out while watching from the sidelines. Yet those experiences never diminished his determination to pursue the things he loved. Music became one of those passions. What started as a childhood joke between Iván, his brother and grandmother eventually led to a lifelong love of guitar. Today, nearly 20 years after forming friendships through music, he still gets together with friends to play and share the songs that brought them together. Throughout his journey, Iván has been surrounded by unwavering support from family and friends. Over time, living with MPS also changed the way Iván viewed those closest to him. Seeing how much his parents worried and cared for him gave him a deeper appreciation for their love and sacrifices. “I am deeply grateful to my family because they have always been there for me throughout my journey," he says. "I never felt alone or abandoned in any way.” If there is one message Iván hopes to share, it is that limitations are part of life, but they are not the whole story. Many barriers come from fear or doubt more than anything else. By recognizing his limits and still choosing to move forward, Iván has learned to create his own path. “It’s not always possible to do everything in the same way as others,” he says. “But it is always possible to find a way, and that is enough.” #FacesOfMPS

Our team had a great time joining more than 500 members of the phenylketonuria (PKU) community from across the United States at the 2026 NPKUA Community Conference in Illinois. The BioMarin team in attendance spent the weekend connecting with individuals living with #PKU, caregivers, advocates, clinicians and researchers while participating in important conversations about the daily realities of the condition and the future of care. We were proud to sponsor the Teen Track, which included social outings, on-site activities, a Teen Zone room and sessions where young people shared their experiences living with PKU. Thank you to the National PKU Alliance staff and volunteers who helped facilitate the teen experience. We would also like to congratulate Amy Oliver on receiving a special award at the event recognizing her many contributions over the years. It was truly moving to hear her daughter, Claire, speak about her work. We add our deepest appreciation for all Amy has done to advocate for the community and successfully change policies to better support people living with PKU. We're grateful to the NPKUA for hosting #NPKUA2026, and to everyone who stopped by to share their experiences and perspectives with us. We are honored to have worked alongside the PKU community for more than 20 years and look forward to continuing to support and learn from you.

At BioMarin, we believe in supporting opportunities that foster connection, accessibility and belonging among the communities we serve. That’s why we’re proud to sponsor the National Adaptive Soccer Camp, which is coming to Seattle from Aug. 28 to 30 to offer adaptive soccer players across the Pacific Northwest and beyond the opportunity to hone their skills, gain confidence and build community in a supportive and competitive environment. Hosted by the United States Adaptive Soccer Association, these clinics are designed for both beginner and developing soccer players, with openings still available for people with dwarfism, lower limb differences or cerebral palsy. Learn more and register to attend by July 31: http://ms.spr.ly/6047v4LhD For questions, reach out to info@unitedadaptivesoccer.com.

Disability inclusion advocate Alycia Anderson joined us at our San Rafael headquarters this week for a special event honoring Disability Pride Month. Invited to speak by our People with Disabilities (PwD) Employee Resource Group, Alycia delivered an insightful talk focused on accessibility, allyship and everyday actions that help build more inclusive spaces. The former technology executive and lifelong wheelchair user shared perspectives shaped by her lived experience on challenging assumptions about ability, fostering belonging, and recognizing accessibility as a shared responsibility and benefit to all. “Our differences are powerful,” says Alycia, who hosts a podcast called Pushing Forward with Alycia. “Inclusion begins when we stop making assumptions about what people can or cannot do.” As we approach the anniversary of the Americans with Disabilities Act on July 26, Alycia’s message reinforced that inclusion is not only about compliance – it is about culture, connection and making sure every person has the opportunity to participate fully. Thank you to Alycia, her husband Marty, our PwD team, and all who joined the conversation and celebration that followed. Learn more about Alycia and her story: http://ms.spr.ly/6049v2mec

“I never want to change who I am. I want the way the world reacts to my differences to change.” For years, Becky Curran has used her voice to challenge assumptions, lead with kindness and address systemic barriers for the disability community. From delivering two TEDx talks to advocating for authentic representation in the media, Becky’s work as a disability inclusion advocate is deeply rooted in her lived experience. Following the tragic death of her infant son Jackson in 2022 due to an avoidable medical error, Becky found a renewed fire to create a safer, more inclusive world. She now honors his memory by carrying his name as her middle name. As part of our #MakingSpace campaign, we sat down with Becky to discuss how she navigates a world not designed for little people, the power of asking "How can I best support you?", and why true inclusion requires curiosity, respect, and accountability. Read more: http://ms.spr.ly/6046vFXKp

We announced that the U.S. Food and Drug Administration has accepted BioMarin's supplemental New Drug Application for full approval of our medicine for children with achondroplasia. The submission is supported by results from three ongoing long-term extension clinical trials, including the largest body of efficacy and safety data of any medicine studied in achondroplasia. Learn more: http://ms.spr.ly/6042v0ZF6

Three episodes, two hosts and one question: Why Do I Pheel This Way? In this video podcast, we passed the mic to co-hosts Connor and Londyn to hear what it’s like to navigate phenylketonuria (PKU) through school, sports, friendships and the everyday moments that shape growing up. A special thank you to Connor and Londyn for bringing honest, relatable voices to the conversation and offering a real-life look at PKU from the people living with this rare metabolic condition. Watch the full three-part series here: https://lnkd.in/eUv2GMJd