ultragenyx.comUltragenyx—Treatment of Rare and Ultrarare Genetic Diseases

Patient Advocacy U.S. ARGENTINA BRAZIL CANADA (English) CANADA (Français) CHILE COLOMBIA Germany ITALY JAPAN MEXICO PERU UNITED KINGDOM UNITED STATES Home Ultra-Committed To transform the lives of as many people living with rare diseases as possible, we dedicate ourselves every day to the development and delivery of treatments of rare diseases where none currently exist. Ultra-Committed Learn more Leadership Access to Our Therapies Early Access Corporate Responsibility Grants & Contributions Patient Advocacy Research We develop therapies for rare and ultrarare genetic diseases by exploring an array of drug modalities to select the best treatment strategy for each genetic disease we are studying. Research Learn more Our Science Our Gene Therapy Platform Our Pipeline DTX401 for GSDIa DTX301 for OTC Deficiency UX701 for Wilson Disease UX143 for OI GTX-102 for AS UX111 for MPS IIIA UX055 for CDD Our Clinical Trial Transparency Commitment Medicines Partnerships Join Our Team We have built a talented team and a vibrant, people-first culture. We share a passion for helping those living with rare diseases. Join our team Learn More Culture Benefits Current Opportunities Internships, Co-ops & Postdoctoral Programs Contact Us Newsroom Access news releases, presentations, and blog posts to follow the latest developments at Ultragenyx. Newsroom Learn More Press Releases Blog Media Asset Library Investors Patient Advocacy U.S. ARGENTINA BRAZIL CANADA (English) CANADA (Français) CHILE COLOMBIA Germany ITALY JAPAN MEXICO PERU UNITED KINGDOM UNITED STATES Menu Close For Avery and Addison, we go beyond every day Advancing medicines for rare diseases Learn more Facebook Linkdin Twitter #GoingBeyondEveryDay Avery and Addison are living with osteogenesis imperfecta. 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 Leadership Our team of passionate biopharmaceutical leaders combines deep expertise in drug development and commercialization Meet our leadership team Pipeline We have one of the largest and most diverse pipelines in rare disease Explore our pipeline Careers Join our talented team Browse current opportunities Latest news View more newsMay 07, 2024 Ultragenyx to Participate at Bank of America’s 2024 Healthcare Conference Read press release May 02, 2024 Ultragenyx Reports First Quarter 2024 Financial Results and Corporate Update Read press release April 30, 2024 Ultragenyx Issues 2023 Corporate Responsibility Report, Showcasing Commitment to Improving Equity and Access to Innovation in Rare Disease Read press release Rare perspective Insights and information on our approach to collaborating with rare disease communities, supporting our teammates at Ultragenyx, and developing new therapies for rare disease Read our blog© 2023 Ultragenyx Pharmaceutical Inc. All rights reserved. PATIENT ADVOCACY CONTACT US PRIVACY POLICY COOKIE POLICY SITE MAP Facebook Linkdin Twitter This site uses cookies to provide you with a more responsive and personalized service. By clicking Accept” below, you agree to the use of cookies on this site. Please read our Cookie Policy and Privacy Policy for more information on the use of cookies on this website. Accept Do not...