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

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.

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 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

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 .

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

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.

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.

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

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

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 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

' #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 #NewbornScreening AwarenessMonth

#NewbornScreening (NBS) alone is insufficient to keep pace with today’s science. Read why political leadership is needed to supplement NBS with #GeneticTesting and help advance the Precision Medicine Answers for Kids Today Act: ultragenyx.co/45RMTXr

This year marks the 60th anniversary of the first mandated #NewbornScreening (NBS) test. Notwithstanding this tremendous progress, we have to adapt the NBS system to keep up with the rapid pace of innovation. Learn more: ultragenyx.co/NBS
#NewbornScreening AwarenessMonth

#NewbornScreening is a vital public health program that saves lives. Watch our CEO, Dr. Emil Kakkis, talk about why we must expand the number of diseases covered so that we can improve outcomes for even more patients: ultragenyx.co/NBS #NewbornScreening AwarenessMonth

Today we announced we have begun dosing the second dose-escalation cohort in our pivotal Cyprus2+ study investigating a potential treatment for #WilsonDisease . Read more about this milestone: ultragenyx.co/459DRV8

Our team attended the International Metabolic Conference this weekend alongside our colleague, Ali Skrinar, who discussed the ways Ultragenyx and the Long-chain Fatty Acid Oxidation Disorder (LC-FAOD) community are allies in advancing research. Thank you, Ali and team!

Today there are opportunities to treat ultra #RareDiseases . Our CEO, Dr. Emil Kakkis, is raising awareness for better policies allowing for the development of treatments that can improve the lives of people with lesser-known diseases that often go unnoticed.