Members of our team were in DC during #RareDiseaseWeek to engage with policy makers and other industry leaders regarding the drug development process and how to make it work better for rare disease patients and their families. We applaud all of the rare disease patients, families and allies who were in DC last week making their voices heard and we stand with the 300 million people worldwide living with rare diseases. National Organization for Rare Disorders EveryLife Foundation for Rare Diseases
I know that view! 😄 Hope you and team had a wonderful and impactful trip Betsy Ricketts Lisa Kahlman! Thanks for the important work you all do!
As a Medical Affairs physician, I appreciate Ultragenyx's proactive work with policymakers to streamline rare disease drug development. Your collaboration with patient advocates is key to driving meaningful policy changes and accelerating innovative treatments during challenging times. Ultragenyx
Thank you to Emil Kakkis and the incredible team at Ultragenyx for your tireless efforts to advocate and develop therapeutics for the rare disease community.
At the Cure GM1 Foundation, we're hoping there will be bipartisan support for the renewal of the PRV ASAP.
A special thanks to those at Ultragenyx working with Saint Louis University's Center for World Health and Medicine to advance FSHD research.
Great photo Emil Kakkis Betsy Ricketts Lisa Kahlman Cheers and keep fighting for rare patients. All the best!
Looking great Lisa!