Alliance CEO Sue Peschin spoke at #WorldOrphanUSA on the patient impact of FDA's Accelerated Approval Program alongside experts from EveryLife Foundation, Johnson & Johnson, and The Center for Medicine in the Public Interest.

#WorldOrphanUSA #rarediseases #rarediseasecommunity

Stay up-to-date on #NewbornScreening progress for #DMD  and hear from experts Paul Melmeyer, #MDA , Lauren Stanford, Parent Project Muscular Dystrophy (PPMD), Annie Kennedy, EveryLife Foundation, & Jill Castle, Little Hercules Fdn. Register today for the webinar on Friday, May 3 at 1pm ET: parentprojectmd-org.zoom.us/webinar/regist…

Today's #WorldOrphanUSA Panel Discussion:

'We need to make sure we have the evidence collected early within the clinical trial ecosystem that then is available to payers to support access at the time of approval' - Annie Kennedy

The EveryLife's Jamie Sullivan and Julie Breneiser from Gorlin Syndrome Alliance discuss 'Patient-first innovation: FDA shifting rare diseases endpoints' #WorldOrphanUSA

Closing this morning's sessions at #WorldOrphanUSA discussing 'Challenge and Opportunity with the Current Regulatory and Legislative Landscape in the US.' The EveryLife's VP of Policy Jamie Sullivan, was joined by Rachel Smith, Mark Trusheim, Kinnari Patel and Peter Marks.

We're Hiring!
Join our team in making a significant difference in the lives of over 30 million Americans affected by rare diseases. We're looking for: Vice President of Individual Giving, Events Manager and Communications Intern.
Interested? Apply here: everylifefoundation.org/careers/

We are thrilled to have our very own Annie Kennedy share the stage with U.S. FDA Commissioner Robert Califf at this year’s #WorldOrphanUSA discussing 'Charting the path forward: Regulatory innovations and orphan drug designations in rare disease development'

Patient experience data has always been critical for FDA, we just didn’t know how to talk about it until recently. The science of #patientexperience data is coming along, particularly with #digitalhealth
- U.S. FDA R. Califf discussing reg innovations w/ EveryLife Foundation A. Kennedy

8 days to go! Get excited for spectacular sessions, including the Keynote Fireside Chat: 'Charting the path forward: Regulatory innovations and orphan drug designations in rare disease development', with...
⭐️ U.S. FDA
⭐️ EveryLife Foundation

Register here: tinyurl.com/ytf27dud

Rare disease advocates looking for personalized coaching, guidance on setting advocacy goals, or assistance in forging connections with legislators- Don't miss this opportunity to enhance your efforts & make a lasting impact!

To apply and learn more visit hubs.li/Q02sB7RQ0

Exciting opportunity from EveryLife Foundation: The #RareIs Scholarship Fund!

To learn more about the scholarship and how to apply visit everylifefoundation.org/rare-scholarsh…

Welcome Michael Pearlmutter, the new CEO of the EveryLife Foundation! 🎉

'As someone who has personally navigated the challenges of a rare blood disorder, I am deeply motivated to harness our collective power to make a significant impact'

Read more: bit.ly/3PGvkUl

Fireside Chat highlight: 'Patient-first innovation: FDA shifting rare diseases endpoints' with:

Julie Breneiser, Executive Director, Gorlin Syndrome Alliance
Jamie Sullivan, Senior Director of Public Policy, EveryLife Foundation

Register for #WorldOrphanUSA 👉 tinyurl.com/ytf27dud

Congratulations to the #MLD community on reaching this significant milestone representing the dedication and sacrifices of numerous individuals! 🙌

We were honored to be at the RDLA @EverylifeOrg #RareDisease Congressional briefing. Thank you for all your efforts to amplify the voices of patient communities, including #BarthSyndrome , and show that we are #NotTooRareToCare about! #RareDisease Day #RDW2024 #RareDC2024

If you’re attending #RDDNIH in person, check out the exhibits from Rare Diseases Clinical Research Network, NORD, EveryLife Foundation, Global Genes, GARD, CURE ID and others! You also can explore scientific posters, art and film from the #RareDiseases community!