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"#NewbornScreening Saves Lives" is not just a slogan. It’s a fact. Dr. Mei Baker, shares why newborn screening is critical to providing the best possible life for newborns. Learn more: ultragenyx.co/NBS #NewbornScreeningAwarenessMonth

Ultragenyx is a strong advocate for #NewbornScreening. Lisa Kahlman supportsour dedication in expanding the number of diseases included on state screening panels. Learn more: ultragenyx.co/NBS

On Monday October 16th, our leadership team and external experts will share an update on Ultragenyx’s development pipeline including our #AngelmanSyndrome and #OstegenesisImperfecta programs. Watch live: bit.ly/3tlGOnL

Ultragenyx to attend #ASBMR2023 to share the latest advancements from our ongoing late-stage program for #OsteogenesisImperfecta (OI). Read more: ultragenyx.co/3Q8C0es

Join us virtually on Monday, October 16 at 8:30 AM ET to hear from two researchers leading our #OsteogenesisImperfecta program during #AnalystDay. Watch the live webcast here: bit.ly/3tlGOnL

Ultragenyx announces new data from the investigative Phase 2/3 Orbit study of setrusumab (UX143) in #OsteogenesisImperfecta (OI) at #ASBMR23. Learn more: ultragenyx.co/3txZhxA

Thank you to Elizabeth Berry-Kravis, M.D. Ph.D., RUSH Medical College, for providing perspective on the clinical meaningfulness of our Phase 1/2 investigational drug for Angelman syndrome at Analyst Day. She spoke with Kemi Olugemo, M.D., VP of clinical development at Ultragenyx.

That's a wrap for our Analyst Day! We shared an update on investigational therapies for #OsteogenesisImperfecta (OI), #AngelmanSyndrome, #WilsonDisease and our gene therapy pipeline. Thank you to the #RareDisease families and communities for supporting our development programs.

Join our diagnostics and public affairs leaders, Nicole Miller and Lisa Kahlman at the North America Rare Disease Summit hosted by Bamberg Health for a discussion on the role of AI, challenges and advances in #RareDisease diagnosis and treatment. Register: bit.ly/406QQ8z

Today at the North America Rare Disease Summit 2023 (#NARDS), Ultragenyx team member Lisa Kahlman, executive director, public policy & public affairs, interviewed Dr. Peter Marks on his view of leveraging the Accelerated Approval pathway in #RareDisease.

At the North America Rare Disease Summit 2023 (#NARDS) last week, Charlene Son Rigby, CEO of Global Genes, discussed the high cost of delayed diagnosis in #RareDisease. Read the EveryLife report here: bit.ly/47fp1NB

We are pleased to announce new positive expansion data from the Phase 1/2 study of GTX-102 for the treatment of #AngelmanSyndrome. Read the full press release: ir.ultragenyx.com/news-releases/…

At #WorldOrphanUSA, our CEO, Dr. Emil Kakkis, will take the stage to deliver a keynote presentation on using biomarkers for #RareDiseases. Nicole Miller, VP of Molecular Diagnostics, will also take part in a panel on #NewbornScreening and the future for rare disease. More below.

Shout out to the 250+ participants who made the #WorldOrphanUSA Congress a memorable event. Our CEO, Dr. Emil Kakkis, inspired us all to unite as one rare nation and advocate for the FDA's accelerated approval of rare disease #biomarkers. World Orphan Drug Congress USA

In Massachusetts, we understand the crucial link between the life sciences and our innovation economy. It’s about people working hard to discover new knowledge, and to turn that knowledge into cures that bring life and hope to the world. Congratulations to the Ultragenyx team

Privileged to host ribbon-cutting for our Somerville R&D facility with @massgovernor, Mayor Katjana Ballantyne, Kate Walsh, and sannstanton! Thrilled to join the Mass. Innovation & Life Science community and advance gene therapy for #RareDiseases. Thank you for joining us!

We're looking forward to attending the #JPM Healthcare Conference next week. Our CEO Emil Kakkis will be presenting on Monday at 10:30 AM PT. Please join us live or virtual: ultragenyx.co/4gNU6NG #JPM2025

Thanks to the White House Office of Science & Technology Policy team for hosting an engaging forum on cell and gene therapy. Our CEO Emil Kakkis and VP policy and government affairs Betsy Ricketts represented Ultragenyx to discuss patient access, affordability, manufacturing and capital investment in CGT. #whitehousecgt

Our CEO Emil Kakkis was joined by our CFO Howard Horn and CMO Eric Crombez on stage at #JPM2025 for our corporate presentation, highlighting exciting milestones ahead. Read more about our financial highlights and updates across our pipeline programs here: ultragenyx.co/40upQSh

With 95% of rare diseases lacking a treatment, research and development can be a challenge —and an opportunity. Ultragenyx is working to relieve families from the struggle of finding the treatment that will transform their child’s life. We can all get behind that #RareDiseaseDay