Ultragenyx

05/12/2026

Today, at the San Francisco Business Times Bay Area Biotech Forum, our CEO and president Emil Kakkis was presented with the first ever Legacy Award by Ron Leuty. He was joined by Ryan Dant, whose story is at the center of why we exist.

Ryan was diagnosed with MPS I as a child, a fatal disease with no approved treatments at the time. With support from Ryan's family and the foundation they built in his name, Emil developed the enzyme replacement therapy that would go on to save Ryan's life, and change the future for individuals diagnosed with MPS I.

Receiving this recognition alongside Ryan is a reminder of how powerful it is when researchers, families, the biotech industry and regulators work together to change the future. We're honored to carry this legacy forward through our work to develop and deliver first-ever therapies for patients and families impacted by rare and ultra-rare diseases around the world.

05/07/2026

Rare disease science has advanced in extraordinary ways, but for many families, the hardest part is still turning that progress into real treatments. Too often, when no one is developing a therapy for their loved one, families are left to find a way forward on their own.

That’s why Rare Bootcamp exists — to help families, advocates, and innovators better understand the path from a scientific idea to a new therapy and make that journey a little easier to navigate.

Learn more about Rare Bootcamp and the families working to change the course of their children’s lives here: https://www.cnbc.com/2026/05/07/rare-disease-families-research-drug-development-ultragenyx.html

Thank you to the Rare Bootcamp sponsors who made the April event possible: Alexion Pharmaceuticals, Inc., Alnylam Pharmaceuticals, BioMarin Pharmaceutical Inc., BridgeBio, Chiesi Global Rare Diseases, Dash Bio, EveryLife Foundation for Rare Diseases, GeneDx, Global Genes, Goodwin, iXCells Biotechnologies, Morgan, Lewis & Bockius LLP, NeuCyte, and Rentschler Biopharma.

04/10/2026

Each Rare Bootcamp session, we’re reminded of the resilience and determination of the rare disease community. Thank you to our co-host, EveryLife Foundation for Rare Diseases and our sponsors.

This week, the EveryLife Foundation for Rare Diseases was proud to co-host the 2026 in Boston, MA, in partnership with Ultragenyx, bringing together advocates, families, and leaders across the rare disease community for three days of immersive learning and connection.

Through expert-led discussions and collaborative conversations, attendees gained practical guidance, resources, and strategies to help move rare disease drug development efforts forward. EveryLife Foundation CEO, Michael Pearlmutter, spoke during yesterday's event.

Each Rare Bootcamp is a reminder of what’s at stake for rare disease families and shows the power of collaboration in pushing the boundaries of what’s possible. Thank you to our sponsors, Ultragenyx, Goodwin, Chiesi Global Rare Diseases, Rentschler Biopharma, Alexion Pharmaceuticals, Alnylam, BioMarin, BridgeBio, GeneDx, Dash Bio, iXCells Biotechnologies, Morgan Lewis, Neucyte, and Global Genes for making this event possible!

02/04/2026

Breaking rare disease news: With the Mikaela Naylon Give Kids a Chance Act signed into law, the Rare Pediatric Disease Priority Review Voucher (PRV) program has been renewed through September 30, 2029.

The PRV program has played an important role in accelerating the development of treatments for children with rare diseases. Its renewal reinforces the path from early research to real options for patients and families, while providing incentives that can help smaller and emerging companies advance promising development programs.

Thank you to the congressional champions including the sponsors, the entire House and Senate leadership for enabling this progress!

We are encouraged by this continued commitment to rare pediatric disease innovation and remain focused on what matters most, delivering meaningful therapies to patients and families who cannot afford to wait.

02/01/2026

Today is Creatine Deficiency Day. Like many rare diseases, early diagnosis and intervention can make a real difference.

Throughout the day, the Association for Creatine Deficiencies is highlighting the journeys of families living with Cerebral Creatine Deficiency Syndrome (CCDS), including early signs to look out for. Follow along to hear their stories.

12/30/2025

Managing Glycogen Storage Disease Type Ia ( ) is a 24/7 responsibility for individuals and families. The constant vigilance and risk of life-threatening low blood sugar take a heavy physical and emotional toll, underscoring the urgent need for better treatment options.

Today we announced that we have completed the rolling submission of our Biologics License Application (BLA) to the U.S. FDA for our investigational gene therapy for GSDIa.

We look forward to keeping the community updated as we are able.

12/29/2025

Today we announced results from the Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta (OI). Neither study achieved statistical significance against the primary endpoints of reduction in annualized clinical fracture rate compared to placebo or bisphosphonates, respectively.

To learn more about the results, including next steps, please read our update to the OI community: https://ultragenyx.com/OI_update_Dec2025

06/16/2025

By building meaningful connections with rare disease patients and families, we are better equipped to develop therapies that prioritize their unique needs. Our CEO Emil Kakkis shares how this commitment shapes our approach to patient-focused treatment development.

We’re proud to bring this perspective to this week as we connect with others who are pushing the boundaries of what is possible for patients.

05/06/2025

Happy ! Today, we’re joining the global rare disease community to raise awareness for Osteogenesis Imperfecta (OI), also known as "brittle bone disease," a rare genetic bone disorder present at birth.

Our team is proudly wearing yellow, a color symbolizing optimism and positivity, to celebrate the strength and resilience of individuals living with OI.

04/22/2025

As we celebrate 15 years of Ultragenyx, our commitment to transforming the lives of families impacted by rare diseases is as strong as ever. With four medicines approved for five diseases and the largest clinical pipeline in rare disease, our team remains relentless in delivering more progress in innovative drug development, more policy change, and more hope for more families.

Hear from our founder, president, and CEO Emil Kakkis as he reflects on our first 15 years.

04/09/2025

Thanks Foundation for Angelman Syndrome Therapeutics and Angelman Syndrome Foundation for leading this important forum. We were inspired by the overwhelming number of advocates who raised their voice to highlight the challenges and unmet needs of families and express optimism for the future as important research progresses forward.

👐 Thank you to those who attended the Angelman syndrome EL-PFDD meeting yesterday.

The meeting recording is now available, and comments can still be submitted so please participate if you have not yet.

⏯️ Watch the meeting: https://buff.ly/i6xoa9x