Carolyn Wang
@cbwang
SVP Global Corp Comm at Ultragenyx, @JohnRitterFdn board member - also mother, beekeeper. I love science, curiosity and agents of change.
Dit vind je misschien leuk
Bernie Sanders and AOC do a joint video explaining their view of the shutdown.
We have people in power making broad claims around certain drugs and autism that have been continuously disproven. The degree to which those comments can undermine public health, do harm to women who are pregnant, create anxiety for parents who do have children who are autistic –…
I'm on a call tonight called White Women: #AnswerTheCall2024 in support of Kamala. First off, there's a lot of us white women on this call... I cannot imagine what is happening over at @Zoom offices right now. I hope someone is delivering them food and liquids. You rock @Zoom!
Kamala just broke Zoom again. Tonight's Zoom to support Kamala Harris is now the first EVER IN HISTORY to have over 100,000 participants. #AnswerTheCall2024
Ultragenyx $RARE gene therapy for rare liver ailment succeeds in late-stage trial statnews.com/2024/05/30/ult… via @Jasonmmast
statnews.com
Ultragenyx gene therapy for rare liver ailment succeeds in late-stage trial
Ultragenyx said its gene therapy for a rare liver ailment, glycogen disease type 1A, succeeded in a Phase 3 trial, setting up a potential approval.
Come and knock on our door, Joyce DeWitt and Priscilla Barnes! It was a Three's Company reunion at our Evening from the Heart event and @gma captured it: abcnews.go.com/GMA/Culture/th… #AortaEd #AortaSupporta #AorticDissection
abcnews.go.com
'Three's Company' stars have mini reunion at benefit for John Ritter: See the photos
"Three's Company" stars Joyce DeWitt and Priscilla Barnes reunited this week at a benefit for late co-star John Ritter.
Thank you to Kaley Cuoco and the rest of the 8 Simple Rules family for supporting our Evening from the Heart last week! Kaley discusses one of her favorite memories of John with @people: people.com/kaley-cuoco-ho… #AortaEd #AortaSupporta #AorticDissection
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.
Company News: @ultragenyx's Emil Kakkis on this week's Angelman syndrome data, rare disease treatment modalities, and more. Full video: biotechtv.com/post/ultrageny…
Ultragenyx reports early success with Angelman syndrome drug, backing up 2022 acquisition statnews.com/2024/04/15/ult… via @ADeAngelis_bio for @statnews
statnews.com
Ultragenyx reports early success with Angelman syndrome drug, backing up 2022 acquisition
Ultragenyx has found early success with its experimental treatment for the rare disease Angelman syndrome.
This workshop w/ participation from @US_FDA will drive an important discussion on applying the Accelerated Approval pathway for #rarediseases and will focus on #Sanfilippo Syndrome. There are a number of novel treatments on the line and many lives at stake. @ultragenyx
Our "Qualifying Biomarkers to Support Rare Disease Regulatory Pathways" hybrid public workshop agenda features over 15 experts & thought leaders from academic research, patient advocacy, drug development, & the @US_FDA. View the full agenda & register: reaganudall.org/news-and-event…
"There are so many rare diseases that have no treatments at all," says @ultragenyx CEO Dr. Emil Kakkis, who says his company has been working to improve the process for the latest precision gene therapies, and is hopeful the FDA may use accelerated approvals for treatment.
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.
I love the Dr. Peter Marks @US_FDA truth bombs, and there were many today at #NARDS. We're closing on a good one: "Genomic medicine is here to stay. It's about [what country] will get the components together first to make it possible." (here with @Penn ODC Dir. James Wilson, MD)
![cbwang's tweet image. I love the Dr. Peter Marks @US_FDA truth bombs, and there were many today at #NARDS. We're closing on a good one: "Genomic medicine is here to stay. It's about [what country] will get the components together first to make it possible." (here with @Penn ODC Dir. James Wilson, MD)](https://pbs.twimg.com/media/F99HcaWXIAAZnGj.jpg)
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
For 20 years The JRF has worked to spread awareness of aortic health. Watch our video to learn more about the latest research developments and how you can help spread awareness of aortic dissection and save lives. youtu.be/oKxAExRX_us
youtube.com
YouTube
20 Years of JRF - The Ritter Effect
Today, we celebrate the opening of our #GeneTherapy manufacturing facility in Bedford, MA, which will enable us to bring cutting-edge gene therapies to transform the lives of people living with #RareDiseases. Learn more: ultragenyx.co/3PG13Wz
In partnership with @MereoBiopharma, we’re pleased to share data from the dose-selection Phase 2 portion of our pivotal Orbit study for patients living with #OsteogenesisImperfecta (OI). Read the press release for more details: bit.ly/3CbO7PZ
The Orphan Drug Act was an important contribution for people living with #RareDiseases, but today many people living with ultrarare diseases are being left behind. Our CEO, Dr. Emil Kakkis, shares why it's time for an #ODA 2.0 to help support the ultrarare disease community.
Why are #RareDisease families being forced into drug development? Thank you @Jared_Whitlock for highlighting the impact of the regulatory environment and "marginal monetary incentives" on critical therapeutic programs. We can do so much better for these rare communities.
My latest is on drugmakers shelving many potential therapies that were seeded by families. For the piece I spoke with families + reviewed public filings/news releases to show the scope of the issue. endpts.com/families-raise…
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