At BioMarin, we are driven by our purpose: to be the biotech leader that translates the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The BioMarin 8 – our core Operating Principles – provide the framework for how we work together every day to fulfill that purpose.

Today we provided an update on our clinical program for Noonan syndrome. Read more: ms.spr.ly/6012abonG

We’re excited to welcome Robert Plenge to our Board of Directors. Robert, who currently serves as Executive Vice President, Chief Research Officer and Head of Research at Bristol Myers Squibb, brings more than 25 years of experience across academia and biopharma and will provide an important scientific perspective to our Board as we build on our research capabilities and expand our pipeline. Read more: ms.spr.ly/6016abB5a

Today we announced new Phase 3 data in children living with hypochondroplasia, which were published in NEJM Evidence and featured in a late-breaking oral presentation at #ESPE2026. These findings reflect our continued commitment to advancing research in skeletal conditions and deepening scientific understanding of #hypochondroplasia, a form of skeletal dysplasia that can impact growth, physical function and everyday life for children and families. Learn more: ms.spr.ly/6011akPav

We’re excited to introduce the BioMarin Skeletal Dysplasia (BISK) Research Awards, a new opportunity to advance investigator-initiated research focused on improving care and outcomes for people living with #skeletaldysplasia. This program supports innovative, non-interventional studies that address gaps and barriers in care. Areas of interest include generating real-world data, patient-reported outcomes, quality-of-life evidence, and qualitative research such as surveys and interviews. Eligible applicants are healthcare professionals, such as physicians, allied health practitioners and research scientists, working within the skeletal dysplasia space in the United States. Letters of intent are due by Wednesday, Sept. 30. Learn more and apply: ms.spr.ly/6013aTiF3 #RareDiseaseResearch #ClinicalResearch #MedicalResearch

We have completed our previously announced agreement to acquire Alesta Therapeutics. The acquisition will strengthen our skeletal conditions portfolio by adding ALE1, an oral small molecule for the potential treatment of hypophosphatasia currently being studied in a Phase 1/2a clinical trial. Read more: ms.spr.ly/6014aTTx8

Today we announced a global settlement with Ascendis Pharma A/S, under which Ascendis will pay royalties to BioMarin on Yuviwel sales in the U.S., EU, Brazil and South Korea. Read more: ms.spr.ly/6011arnnT

A global panel of experts has developed the first international consensus recommendations to help enable earlier and more accurate diagnosis of hypochondroplasia. Published in Nature Reviews Endocrinology, the recommendations provide a comprehensive framework that integrates clinical, radiographic and genetic assessments, aiming to reduce diagnostic uncertainty and enable more timely, consistent care. We were proud to support this initiative as part of our commitment to improving recognition and diagnosis of hypochondroplasia around the world, and we look forward to working with medical communities to ensure these guidelines are implemented. Read more: ms.spr.ly/6018auDwI

Today we announced that we have entered into a definitive agreement to acquire Alesta Therapeutics to gain Alesta’s lead clinical stage asset, ALE1, an orally active, small molecule for the potential treatment of a rare genetic bone condition called hypophosphatasia (HPP). ALE1 has the potential to be the first oral therapy for HPP and is currently being evaluated in an ongoing Phase 1/2a clinical trial. Read more: ms.spr.ly/6041az85H

Cristin Hubbard is still motivated by the same idea that sparked her earliest interest in healthcare and eventually led her to biopharma: getting medicines to the people who need them most. At BioMarin, Cristin says she feels the impact of that commitment as strongly as ever. “What I love about working in rare disease is the connection to the communities we serve,” says Cristin, BioMarin’s Chief Commercial Officer. “You feel like every contribution matters so deeply for patients and their families.” From early work as a medicinal chemist to leading global commercial teams, her career has been shaped by a consistent focus on bringing innovation closer to patients. She’s applying those experiences to help BioMarin leverage its global footprint and capabilities and ultimately reach more people living with rare genetic conditions around the world. We sat down with Cristin to reflect on her professional journey, her leadership philosophy and how her experience across a variety of roles has prepared her for this moment at BioMarin. Read more in the full interview: ms.spr.ly/6012aHZPo

Today we announced Q2 2026 results, including a 20% increase in total revenues compared to the same quarter last year. This was driven by our acquisition of Amicus Therapeutics and growth in skeletal conditions and phenylketonuria. We also increased full-year guidance for total revenues, revenue for skeletal conditions and non-GAAP diluted earnings per share. Read more in our press release: ms.spr.ly/6015aEv7L $BMRN

We’re looking forward to sharing an update with investors on our Q2 results Thursday, Aug. 6 at 4:30 p.m. ET / 1:30 p.m. PT. Join our earnings call and webcast here: ms.spr.ly/6013aE8bF

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: ms.spr.ly/6012vCgaa

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: ms.spr.ly/6017v71vB

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: ms.spr.ly/6011v0Z0h

Visali Ramanathan and our Business Development team represented BioMarin at the @IAmBiotech (BIO) International Convention, the world’s largest gathering of the biotech industry, this week in San Diego. Visali provided an overview of our approach to partnering and spoke about the critical role business development will play in accelerating BioMarin’s growth to increase our impact for people living with rare conditions. Interested in partnering with BioMarin? Get in touch here: ms.spr.ly/6018vqeMm

We announced new data at ENDO 2026, the Endocrine Society Annual Meeting, highlighting progress in skeletal conditions research. These updates include findings from an investigator-led study in children with hypochondroplasia and Phase 1 data for our investigational long-acting medicine for children with achondroplasia. Learn more: ms.spr.ly/6017vYM79

Today we announced positive topline results from a Phase 3 pivotal study in children with hypochondroplasia, a genetic skeletal condition for which there are currently no approved treatments. The results mark an important step toward addressing the unmet needs of children living with hypochondroplasia. Learn more: ms.spr.ly/6017vp0dh

Today we provided an update on the Phase 3 ENERGY 3 study evaluating BMN 401 in ENPP1 deficiency. Read more: ms.spr.ly/6019vTKaB

For decades, the National MPS Society has brought families together through conferences, support programs and advocacy initiatives. But as leaders at the organization listened more closely to the community they serve, one reality became increasingly clear in recent years: many families impacted by #mucopolysaccharidosis (#MPS) and #mucolipidosis (#ML) were still not being reached. “We’ve always known the importance of getting families and people together,” says Terri Klein, President and CEO of the National @MPSSociety. “But what if we could bring families together that we didn’t already know – families who may not be members of the Society, who may not be connected in other ways, and who may have more need?” A new approach to community outreach called Crossing Paths emerged as a response to help proactively find families, listen to their realities, and begin breaking down barriers to care and connection. Rather than waiting for families to find the organization, Crossing Paths brings the National MPS Society into neighborhoods and communities across the country where engagement has historically been the lowest, creating welcoming, local gatherings designed to foster trust and understanding. More than three years in, the team has held 18 Crossing Paths events across the country that have helped bring additional families into the fold, driving greater equity and contributing to a more holistic understanding about the needs of the community. “Our biggest learning from Crossing Paths has been that we don’t know what we don’t know. And so we have to listen to the community,” says Leslie Urdaneta, Director of Family Support and Pathways at the National MPS Society. “We have to listen to these families first.” Learn more about Crossing Paths in the second installment of our two-part video series on the National MPS Society’s community programming: ms.spr.ly/6018vuoJ0