Ultragenyx

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

02/27/2025

The community is built on millions of unique experiences. Yet, it’s the shared journeys, common challenges, shared passions and celebrations, and united voices that bring us all together. We're thrilled to announce the launch of UltraAdvocates, a new initiative dedicated to amplifying the voices of rare disease patient and caregiver advocates while collaborating on shared priorities to address challenges and celebrate milestones.

Thank you to our first UltraAdvocates, Amber, Ashley H., Ashley W., and Meri for your strength and inspiration. We can’t wait to create change with you. Read more about our UltraAdvocates initiative: https://ultragenyx.co/3F8fE9D

10/03/2024

Thanks Angelman Syndrome Foundation and Foundation for Angelman Syndrome Therapeutics for hosting this important forum. We look forward to connecting with the community on October 23.

10/03/2024

Thanks Angelman Syndrome Foundation and Foundation for Angelman Syndrome Therapeutics for hosting this important forum. We look forward to connecting with the community on October 23.

03/21/2024

Save the date, March 26th! Ultragenyx is proud to partner with Cure Sanfilippo Foundation and the National MPS Society to host a virtual town hall as part of our commitment to providing timely information to the MPS IIIa community. The session will be moderated by scientific officers Matthew Ellinwood, DVM, PhD, from the National MPS Society, and Cara O’Neill, MD, FAAP, from Cure Sanfilippo Foundation. During this town hall, Ultragenyx’s MPS IIIa investigational UX111 program will be discussed, and questions previously submitted by the community will be answered.

Learn more and register: https://bit.ly/3IGhvB8

10/16/2023

Ultragenyx announces new data from the investigative Phase 2/3 Orbit study of setrusumab (UX143) in (OI) at the American Society for Bone and Mineral Research 2023 Annual Meeting ( ). U.S. residents can learn about trial enrollment at https://www.ultraclinicaltrials.com/

10/04/2023

This month is , and we are honoring those living with rare liver diseases. Glycogen Storage Diseases are a group of rare inherited conditions affecting the body’s glycogen use and storage. Glycogen is complex sugar that serves as a form of energy storage in the body, particularly the liver and muscles. The build up of glycogen can have detrimental effects on organ function, especially the liver. Learn more about the conditions we study at ultrarareadvocacy.com

09/28/2023

: Approximately 4 million babies are screened for certain genetic, metabolic and hormonal disorders? This simple test, known as , can identify serious life-threatening illnesses, allowing doctors to step in and start treatment as soon as possible. Discover why it’s critical to expand the number of diseases screened: https://ultragenyx.co/NBS