Today we reported our second quarter 2023 financial results and corporate update. Read more here: https://lnkd.in/gNvpKPmc #FinancialResults
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Congratulations to our Vice President of Policy, Government and Public Affairs, Betsy Ricketts, for being honored by the San Francisco Business Times as one of the 75 most influential women in the Bay Area. Betsy leads the Ultragenyx policy, government and public affairs team supporting policy change that has had an impact on the entire #RareDisease community including leading initiatives to support the diagnosis of rare diseases, regulatory policy to support rare and ultrarare disease drug development and initiatives to expand patient access to approved therapies. See who made the list: https://lnkd.in/eMQsbBvM #SFBTWomen
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In this new STAT First Opinion piece, our CEO Emil Kakkis urges the #FDA cardiorenal advisory committee to pay attention to the "totality of evidence" when reviewing the Stealth BioTherapeutics investigational therapy for #BarthSyndrome tomorrow.
On Thursday, another #ultrarare therapy will be subject to the gaze of an #FDA advisory committee, with the fate of over 200 boys diagnosed with #Barth syndrome worldwide in the balance. Tomorrow the cardiorenal advisory committee will be reviewing the NDA for Stealth BioTherapeutics investigational medicine. In this @STAT News first opinion piece, I call on FDA to take a “totality of the evidence” approach as the data in long-term treatment and early access treatment have shown important benefit in these patients while also acknowledging that there is no adequate, approved or available therapy in this disease. There is an opportunity here to support a more hopeful future now for the one in a million Barth boys. #RareDisease https://lnkd.in/eY3ZkqtC
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We are thrilled that Dr. Heather Lau MD MS, Executive Director of Global Clinical Development at Ultragenyx, is on the PharmaVoice 100 list of transformative life science leaders. Heather oversees cross-functional clinical teams, collaborates with academic institutions, opinion leaders and regulatory agencies, while ensuring the patient perspective is integrated into every stage of drug development. Her contributions to rare disease push the boundaries of what is possible and have profoundly improved the lives of countless children with #NeurogeneticDisorders and their families. Congratulations Heather! Read Heather’s feature: https://lnkd.in/eqja-Xfq
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Each year, the California Life Sciences (CLS) hosts the Pantheon Awards to recognize those making exceptional contributions to life sciences. We’re thrilled that our Executive Vice President and Chief Commercial Officer, Erik Harris, is a finalist in the Elizabeth Schar Inspiring Future Leaders category. Erik has been instrumental in building and shaping Ultragenyx and its unique company culture. His steadfast commitment to people, allyship, and the rare disease community is evident in everything he does. Cast your vote: https://lnkd.in/ewrA47BB
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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!
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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
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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
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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
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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
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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
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