Living with #GlycogenStorageDiseaseTypeIa (#GSDIa) requires constant vigilance to manage blood sugar levels. Currently, individuals rely on cornstarch and strict dietary management to prevent life-threatening hypoglycemia and even when everything is done right, acute emergencies and chronic health problems affecting the liver, kidneys, bones, metabolism and more, persist. Since 2002, The Children’s Fund for GSD Research has driven progress by raising awareness and funding research for new treatments. Board members Michael Julius and Lisa Hodes stress the urgent need for safe, effective, and accessible treatments. Read more from Michael and Lisa on the future of GSDIa treatments: https://lnkd.in/eHJ3RSD5
关于我们
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
- 网站
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https://www.ultragenyx.com
Ultragenyx的外部链接
- 所属行业
- 生物技术研究
- 规模
- 1,001-5,000 人
- 总部
- Novato，California
- 类型
- 上市公司
- 创立
- 2010
- 领域
- rare disease和ultra-rare disease
地点
Ultragenyx员工
动态
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Thanks, Foundation for Angelman Syndrome Therapeutics and Angelman Syndrome Foundation for organizing the 2nd Annual AS Congressional Advocacy Day! It was great to join the #AngelmanSyndrome community in advocating for patient-first policies. #RareDisease
??? Today marks our 2nd Annual AS Congressional Advocacy Day, where passionate advocates take to Capitol Hill to meet with congressional leaders. ?? Through personal stories and a united voice, we are driving meaningful conversations and making specific legislative requests to ensure Angelman syndrome remains a priority in the U.S. Congress. Our 2025 priorities include: Protecting Medicaid, Credit for Caring Act tax benefit, and supporting AS-specific focus and funding by federal agencies. ?? We urge families, friends, researchers, and clinicians to amplify our voices by reaching out to your congressional leaders in support of the in-person advocacy happening right now on Capitol Hill! ? We’ve made it simple—with just a few clicks, you can send a powerful message to ensure Angelman syndrome remains a priority in Congress. Every email makes a difference! ?? Click here to take action: https://buff.ly/CzOAkBI
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On this #EmployeeAppreciationDay, we want to thank U, our employees, for your dedication and commitment to #GoingBeyondEveryDay to support the #RareDisease community. U consistently help us find a way to do what is right for the patients and families we serve.
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During #RareDiseaseWeek, members of our team attended the Rare Disease Fly-In Lobby Day in Sacramento, Calif. hosted by California Life Sciences (CLS) where Alison Skrinar, our vice president of endpoint development and strategy, gave an update on industry advancements and innovation in the rare disease space. Thank you, California Life Sciences (CLS) for hosting this important event as we jointly advocate for better policies that strengthen our ability to bring treatments for rare and ultrarare disease patients. Julie Boyd Erik Harris Maria Isabel Bueso
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Members of our team were in DC during #RareDiseaseWeek to engage with policy makers and other industry leaders regarding the drug development process and how to make it work better for rare disease patients and their families. We applaud all of the rare disease patients, families and allies who were in DC last week making their voices heard and we stand with the 300 million people worldwide living with rare diseases. National Organization for Rare Disorders EveryLife Foundation for Rare Diseases
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In Massachusetts, we proudly marked #RareDiseaseDay by participating in MassBio’s Rare Disease Day Forum. The event’s MC, Erin Frey, our senior director of state government affairs, brought attention to advancements in the rare and ultrarare disease space. The ‘Possible Talk’ was moderated by Matthew Fuller, our VP, head of gene therapy research, and featured Nathan Guo and Ada Lio, Rare Bootcamp alumni and founding board members of the ZTTK SON-Shine Foundation, who shared their experience forming their foundation and working to develop a treatment for their son, Lucas. Huge thanks to MassBio and all participants for a day filled with meaningful, impactful and actionable discussions! Dennis Huang
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Ultragenyx转发了
In recognition of #RareDiseaseDay, our member companies have been raising awareness of policy issues in Washington, DC. Did you know that 95% of rare diseases lack a treatment, leaving patients and their families without hope? Companies like Ultragenyx are on the front lines of innovation, investing in solutions that larger treatment developers may ignore. “The goal is to relieve families from the challenge of finding the treatment that will transform their loved one’s life,” explained Betsy Ricketts, Vice President, Government and Public Affairs. That’s something we can all get behind.
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#RareDiseaseDay is a reminder of our purpose to transform the lives of people with #RareDiseases. Today, we share our stories with pride and celebrate the patients, families, and communities we serve. Hear from some of our dedicated team members as to why Rare Disease Day is so meaningful to them!
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This week Ultragenyx was recognized in partnership with?Mereo BioPharma?by?EURORDIS-Rare Diseases Europe with the Black Pearl Award for patient engagement.?The Black Pearl Awards celebrate the hard work, innovative thinking, and dedication to the rare disease community.?It was an honour for us to share this moment with?Mereo,?Osteogenesis Imperfecta Federation Europe Board member, Stephanie Claeys,?and?many of our #RareDisease community partners, colleagues and rare disease communities in Europe and beyond.? Jane Cooper?Jennifer McCue #EURORDISAwards2025 #GoingBeyondEveryday?
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The #RareDisease 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://lnkd.in/eJfGxk88 #RareDiseaseDay #ShowYourStripes