Ultragenyx

Our 3rd ESG report is out! We've made a lot of progress over the last year for the #RareDisease community, patients and our employees. See our highlights: https://lnkd.in/emKfNXBc   #ESG #CorporateResponsibility

We couldn’t imagine a better way to ring in Zach’s 15th birthday than with the passage of The Zachary Thomas Newborn Screening Act, HB 77, in Alabama by Governor Kay Ivey. Congratulations to the patient advocates whose leadership made this happen. Swapna Kakani, MPH, Brooke Thomas and the entire AL RARE team, your diligent and long effort to improve #NewbornScreening in Alabama has inspired us all. This year, due to the tireless and persistent effort of coalitions of people who live with and care about #RareDiseases, Tennessee, and now Alabama, have seen great success in achieving Recommended Uniform Screening Panel (RUSP) alignment, bringing the total number of states to 13. Allie Ladd, Terry Jo Bichell and Governor Bill Lee, thank you for your meaningful contributions in Tennessee and for signing this significant legislation into law.   The fight for equitable newborn screening continues. Early detection of rare diseases leads to prompt treatment that can help prevent permanent disability, developmental delay, and death. It’s important for states to screen for all conditions on the RUSP. This enormous impact is what drives our State Government Affairs team to #GoBeyondEveryDay to support RUSP alignment efforts.   #RareDisease #NBS #RUSP 

“The mood now not to pay for accelerated approval drugs is completely wrongheaded. Biomarkers that are evidence of underlying disease are superior to clinical endpoints; these biomarkers are not a compromise. If we design drugs to target the source of disease, we’ll move toward curing the disease rather than simply addressing symptoms. This is where we need to go.” - Dr. Emil Kakkis   This week, our CEO Dr. Emil Kakkis joined a distinguished panel at the Health Technology Innovation Policy Conference 2024 for discussions about the impact of the Inflation Reduction Act on the industry and the FDA’s regulation of biologics. Dr. Peter Marks, the Director of the Center for Biologics Evaluation and Research of the FDA addressed challenges with respect to regulation of cell and gene therapies, and mentioned several things that the FDA hasn’t leveraged to the full extent yet: ➡️ Maximal use of the accelerated approval pathway ➡️ Global harmonization of regulatory requirements to allow commercialization in other countries sooner  ➡️ Faster communication between FDA and sponsors (“warp speed for rare diseases”) to shorten length of development timelines Thank you to Stanford Byers Center for Biodesign for hosting an insightful event, and to Jeff Allen, Peter Kolchinsky, Ken Drazan and Dr. Peter Marks for contributing your expertise and insights. #RareDiseases #HealthcarePolicy #Innovation 

"When we honor our sorrows as much as our joys, our failures as much as our successes, we create a worldview in which every part of us belongs." - Dr. BJ Miller, MD A sincere thank you to Dr. BJ Miller, a palliative care physician who joined the Ultragenyx team this week for our latest employee UltraTalk. In his talk, Dr. Miller explored the concept of happiness as something that is rooted in accepting all parts of ourselves. This acceptance of life’s highs and lows creates internal peace and is the key to true resilience and happiness.

Juliet's story is one of many who are fighting the devasting challenges of #OsteogenesisImperfecta (OI). While Juliet and her family have faced unimaginable challenges both emotionally and physically, they've never given up, and neither will we. On #WishboneDay, we shine a light on these #RareDisease warriors and their families. Read Juliet’s story to learn why we #GoBeyondEveryDay for those living with OI: https://lnkd.in/eDErZ8TW #OIAwarenessWeek

Advocacy leaders Matthew Ellinwood, Chief Scientific Officer at the National MPS Society, and Mark Dant, Executive Director of The Ryan Foundation for Rare Disease Research, wrote an insightful article emphasizing the necessity of policy revisions for #AcceleratedApproval, underscoring the unique nature of #RareDiseases and why they require a distinct approval pathway rather than being lumped into standard processes.   View the article: https://lnkd.in/gCwwwmqy

The academic debate on #AcceleratedApproval in oncology continues - with a publication that addresses the serious lapses of the recent JAMA article from the PORTAL group at Brigham and Women's Hospital. This new study estimates that FDA's #AcceleratedApproval has added roughly 263,000 additional years of life for approximately 911,000 #oncology patients. That sounds like a meaningful outcome for those patients and their families. Read the full article published in the Journal of the National Comprehensive Cancer Network: https://lnkd.in/gyBnWvmV #RareDisease

Today, we reported our first quarter 2024 financial results and corporate update. Read more here: https://lnkd.in/gHQbFnjP   #Ultragenyx #FinancialResults

We’re proud to share that we’ve been awarded the 2024 Top Workplaces Culture Excellence award in recognition of our exceptional achievements in Purpose & Values, Innovation, Compensation & Benefits, Work-Life Flexibility, and Leadership. Fostering a sense of community where every team member can find their place, make a meaningful difference and flourish is a top priority for us. Thank you to all our team members for their contributions in making Ultragenyx a great place to work. Learn more: https://lnkd.in/eunqYyqk #WorkLifeBalance #TopPlaceToWork #WorkplaceCulture

Our 2023 Corporate Responsibility Report is out! The report celebrates the progress across our pillars of innovation, patient support, employee engagement, community impact, environmental stewardship, and governance. Read about the report: https://lnkd.in/eU4jN4r3 #CorporateResponsibility