Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community
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We're fans of the work this East Bay STEM program is doing for future innovators- you go girls!
Thank you, Ultragenyx, for your generous support of our STEM programs for girls during the 2024-2025 school year—together, we're empowering the next generation of innovators!
The FDA granting Breakthrough Therapy designation for our investigational therapy setrusumab is a significant milestone and supports our work to get a new treatment for OI to patients: https://lnkd.in/esS8JACV
Thanks Foundation for Angelman Syndrome Therapeutics and Angelman Syndrome Foundation for hosting this important forum. We look forward to connecting with the community on October 23. #AngelmanSyndrome
The Phase 3 Efficacy and Safety Study of GTX-102 (ASO) in Pediatrics, sponsored by Ultragenyx, is now listed on ClinicalTrials.gov. Interested in learning more? ASF and FAST will be hosting a community webinar about the trial, named Aspire, with the Ultragenyx team on Oct 23, 2024 at 1:00pm EST. Submit your questions and register below. Submit your questions and register below: https://buff.ly/4eIGvFY
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. #AngelmanSyndrome
The Phase 3 Efficacy and Safety Study of GTX-102 (ASO) in Pediatrics, sponsored by Ultragenyx, is now listed on ClinicalTrials.gov. Interested in learning more? ASF and FAST will be hosting a community webinar about the trial, named Aspire, with the Ultragenyx team on Oct 23, 2024 at 1pm EST. Submit your questions and register below. Trial Information: https://lnkd.in/g9jJatJG Webinar registration: https://lnkd.in/gUzBiMxK
We’re looking forward to attending the American Society for Bone and Mineral Research (ASBMR) Annual Meeting in Toronto, Canada from September 27-30. We will be presenting multiple research abstracts on #OsteogenesisImperfecta, including an encore presentation of the 14-month data from our Phase 2/3 Orbit study. Find #ASBMR2024 oral and poster presentation details in our press release: https://lnkd.in/eFwN8nWX
We urge the Senate HELP Committee to pass the #CreatingHopeReauthorizationAct tomorrow to reauthorize the Priority Review Voucher programs as we continue to educate lawmakers on this critical program for #RareDisease drug development. Many thanks to our Executive Director of Public Policy, Lisa Kahlman, for representing us in D.C. this week! Find out more: https://lnkd.in/eWTJKiSK
Amanda lives with a rare genetic disorder called #WilsonDisease. Her daily symptoms can sometimes fluctuate between limited mobility and tremors, but she doesn't let that stop her from pursuing her love of sewing and designing her own fashions. Visit our website to learn more about Wilson disease: https://lnkd.in/drdVhH24 #RareStories
“Newborn screening saves lives" is not just a slogan. It’s a fact. Within 24-48 hours, a baby's life can completely change through early detection of certain diseases that may cause permanent disability, developmental delay and even death. Dr. Mei Baker shares why #NewbornScreening is critical.
Newborn screening (NBS) is crucial for the early detection of certain genetic, metabolic, hormonal or functional disorders. With early detection, infants can receive timely medical intervention that can help prevent permanent disability, developmental delay and death. While #NewbornScreening is vital, there isn’t much known regarding how families experience receiving a positive or uncertain NBS result. We want to highlight an important study currently seeking parents of children who have received a positive or uncertain screening result since May 2021. Find out more: https://lnkd.in/gE8mStpx
The reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program would be a major win for the #RareDisease community. We are one step closer to that outcome as the House Energy & Commerce Committee unanimously passed legislation this week to extend the program through September 30, 2029. We want to give special recognition to the bill’s cosponsors ─ Representatives McCaul, Eshoo, Bilirakis, Barragan, Trahan, and Burgess and their staff ─ along with Chair McMorris Rodgers and Ranking Member Pallone for working together to support the rare disease community. While this is a significant milestone, we eagerly await passage of the legislation in both the House and Senate so that rare pediatric diseases continue to receive the attention they deserve. Learn more about the PRV Program: https://lnkd.in/e4ka-7uY
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