BioMarin Pharmaceutical Inc.

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BioMarin Pharmaceutical Inc.

At BioMarin, we are committed to transforming lives through genetic discovery. At BioMarin, patients are at the heart of what we do.

Applying our knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. The end goal has always been better lives and now we can reach more. And the more people we reach, the more our impact can grow. The BioMarin page is a place to connect with and learn about our company. We will provide company news and updates, as well as share stories from our patients. Our goal is to engage with our community by providing useful and interesting information, and fostering open and respectful dialogues about the diseases we seek to treat. While we are committed to fostering important relevant discussions and respect the rights of everyone to voice their opinions, we ask that users/followers of our social media channels are respectful to other members and follow our Community Guidelines. See our full Community Guidelines here: https://www.facebook.com/BioMarinOfficial/app/145740286161845

10/30/2025

Christina Pao’s passion for science and desire to translate research into clinical practice for genetic skeletal conditions led her to her current role with BioMarin’s Global Medical Affairs team. She and her colleagues are focused on helping the medical community better understand these rare conditions to enable earlier diagnosis and multidisciplinary care.

“Diagnosing skeletal conditions can be quite complex,” Christina says. “In , children may not be diagnosed until they're 4 or 5 years old, and so it's our job to really educate doctors on recognizing those signs and symptoms.”

Christina shares her story in the latest edition of our video series, Signature Science. Highlighting scientists in roles across the company, the videos seek to uncover how our colleagues draw on their unique backgrounds and experiences to make their mark – or, put their own signature – on BioMarin’s breakthrough science.

Read more about Christina’s journey and how she is working to help improve outcomes for people living with genetic skeletal conditions: https://bit.ly/4hABp1b

10/29/2025

Meet Celia, a primary school teacher from Lyon, France who loves art, traveling, laughing with friends and eating Italian cuisine. Celia is also one of the ambassadors sharing her experience living with as part of the social media campaign, created by BioMarin in collaboration with multiple patient advocacy organizations, including APPT - Association des Personnes de Petite Taille.

Through a series of photos, she invites people to view the world from her perspective while doing everyday tasks at home, shopping, socializing and getting around the city. Celia is hopeful that enabling people to see the world from her vantage point will help create more understanding about what it means to live with a skeletal condition.

“Achondroplasia and other skeletal dysplasias are still surrounded by many misconceptions,” says Celia. “To combat stereotypes and foster a more inclusive society, it is essential to improve visibility and understanding.”

We thank Celia for sharing her lived experience with us, and our partners at APPT for collaborating on this campaign.

Learn more: https://bit.ly/42VflrY

10/28/2025

Families, advocates and experts came together in West Palm Beach, Florida, for last weekend’s Little Legs Big Heart Skeletal Dysplasia Conference.

This year’s program featured discussions on the latest research in and , accessible workouts and wellness activities, including a soccer clinic with the U.S. Dwarf Futbol Association and United Adaptive Soccer Association, and a special Camp Ellellbee session. Each moment celebrated learning, creating a space to share lived experiences and strengthen connections across the community during .

Thank you to Kristen DeAndrade and The Little Legs Big Heart Foundation for leading this important work and welcoming us to take part.

10/25/2025

In her first year at Cuesta College in San Luis Obispo, California, Annika Koch is aiming to strike a balance between her academic pursuits and personal interests. She hopes to one day become a nurse, citing her experience growing up with and the healthcare professionals who made an “immeasurable difference” in her life as her inspiration.

As we recognize , we are proud to support people like Annika and share their stories.

Learn more about Annika and the 2025-26 RARE Scholars: https://bit.ly/4nPBOz0

10/23/2025

Like many 4-year-olds, Juliette is bursting with energy. She loves to dig her hands into anything sensory – sand, dirt or the corn pit at the local pumpkin patch, where she could happily sit for hours. Her parents, Joey and Jemma, describe her as quick and determined. If she wants something, she doesn’t wait for help; she gets it herself.

Juliette was diagnosed just over a year ago with mucopolysaccharidosis (MPS) type IIIA, a rare genetic condition that affects approximately 1 in 70,000 children born in the United States. Also known as Sanfilippo syndrome, MPS III is a progressive condition with no approved treatments that leads to a decline in cognitive abilities, loss of motor skills, musculoskeletal issues and, ultimately, premature death before children reach adulthood.

Joey and Jemma began noticing changes about six months ago, including regressions in speech, motor skills and comprehension.

“Juliette used to have a limited vocabulary, and she’d get visibly excited when I got home from work in the afternoon,” says Joey. “About six months ago, though, that all started to change. She’s non-verbal now, and very impulsive.”

Despite these changes, Juliette remains light-hearted and content, rarely getting upset. She is surrounded by a strong support system, including her older sisters, Samantha and Naomi, who remain protective and proud even when Juliette’s needs take precedence. Juliette's grandparents recently moved to town to be closer and more available. Her preschool teachers and aides have created a nurturing environment, and her one-on-one support staff are familiar with Sanfilippo syndrome, offering some comfort in an otherwise uncertain journey.

As for the future, her parents hold onto hope, not just for Juliette, but for all families facing this condition. They hope for quicker diagnoses and a world where kids like Juliette have every chance to grow and thrive.

“A realistic hope is to have a readily available treatment that provides Juliette with some semblance of a typical and much longer life,” Jemma says.

Read more about Juliette’s story on the National MPS Society website: https://bit.ly/47l0gkH

10/22/2025

For Lynsey, advocacy isn’t just a career, it’s personal. Born with arthrogryposis, a rare bone condition, she spent her childhood navigating surgeries and sharing her story with researchers in hopes of sparking progress. Today, as Associate Director of State Government Affairs at BioMarin, she channels that lived experience into powerful policy work that supports rare disease communities, including children living with skeletal conditions.

Whether she’s engaging with legislators, building coalitions or helping lead BioMarin’s People with Disabilities ERG, Lynsey brings determination and empathy to everything she does. Her story is a testament to the impact of lived experience in shaping a more inclusive and hopeful future.

Read more about Lynsey’s path from patient to advocate: https://bit.ly/4hqkMFh

Address

770 Lindaro Street
San Rafael, CA
94901

Website

http://www.biomarin.com/

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