EveryLife Foundation for Rare Diseases

📣 Today, April 1, 2025, the U.S. Department of Health and Human Services (HHS) eliminated thousands of positions across federal agencies including the Food and Drug Administration (FDA), U.S. Centers for Disease Control and Prevention (CDC), the Administration for Community Living (ACL), and The National Institutes of Health (NIH). While full details of the roles and divisions impacted are still being assessed, we know that our landscape has significantly shifted today. 𝗢𝘂𝗿 𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 𝗰𝗼𝗺𝗺𝘂𝗻𝗶𝘁𝘆 𝗶𝘀 𝗶𝗻𝗱𝗲𝗯𝘁𝗲𝗱 𝘁𝗼 𝘁𝗵𝗲 𝗽𝘂𝗯𝗹𝗶𝗰 𝘀𝗲𝗿𝘃𝗮𝗻𝘁𝘀 𝗮𝗰𝗿𝗼𝘀𝘀 𝘁𝗵𝗲𝘀𝗲 𝗮𝗴𝗲𝗻𝗰𝗶𝗲𝘀 𝘄𝗵𝗼𝘀𝗲 𝗲𝘅𝗽𝗲𝗿𝘁𝗶𝘀𝗲, 𝗶𝗻𝗻𝗼𝘃𝗮𝘁𝗶𝗼𝗻, 𝗮𝗻𝗱 𝘃𝗮𝗹𝗶𝗮𝗻𝘁 𝗰𝗮𝗿𝗲𝗲𝗿𝘀 𝗵𝗮𝘃𝗲 𝗳𝗼𝗿𝗲𝘃𝗲𝗿 𝗰𝗵𝗮𝗻𝗴𝗲𝗱 𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲. And while there is still so much we do not yet know, we do know this: 💠 Our community is powered by individuals who each faced an inflection point.  💠 A life-altering diagnosis. 💠 A moment in which our own or a loved one's rare disease changed our story forever. 💠 A moment that could have signified ‘the end’, but for those within our community – it was just the beginning. While the U.S. Department of Health and Human Services (HHS) reorganization is unprecedented, we know we cannot lose our footing. Our community will rebuild and learn to navigate this new landscape. And someday soon, we will transform this into a beginning. United. Amplified. And stronger than ever. We are the rare disease community.

What is YOUR dream? #RAREis Scholarship Fund applications are now open! Thanks to the support of Amgen, The EveryLife Foundation for Rare Diseases established the fund to enrich the lives of adults living with rare diseases by providing support for their educational pursuits. This year, one-time awards of $5,000 scholarships will be provided to 104 rare disease recipients. Applications will close on April 28, 2025. Learn more and apply here: https://hubs.li/Q03dXNhY0

Thank you to the nearly 1,000 advocates who attended Rare Disease Week on Capitol Hill in Washington, D.C. #RareDC2025   Advocates learned about policy proposals impacting the rare disease community and made their voices heard through 363 meetings with members of Congress, representing 184 patient organizations. To everyone who joined us—and to those cheering us on from afar—thank you and see you next year! Watch the #RareDC2025 wrap-up video below and relive the experience. Thank you to our sponsors for their support! Alexion Pharmaceuticals, Inc. Sanofi Amgen CSL Takeda argenx Merck Biogen Travere Therapeutics GSK Harmony Biosciences Genentech Pfizer Johnson & Johnson Mallinckrodt Pharmaceuticals Soleno Therapeutics, Inc. UCB Acadia Pharmaceuticals Inc. Avidity Biosciences, Inc. Amicus Therapeutics BioCryst Pharmaceuticals, Inc. Biotechnology Innovation Organization BioMarin Pharmaceutical Inc. Chiesi Group Faegre Drinker Ionis Pharmaceuticals, Inc. Mitsubishi Tanabe Pharma America Novartis Neurocrine Biosciences Sumitomo Pharma America, Inc. Stoke Therapeutics Otsuka Pharmaceutical Companies (U.S.) Ultragenyx

🎉Virginia Governor Glenn Youngkin approved HB 1782, a bill that mandates the state to consider national recommendations when determining which conditions newborns are screened for at birth. 👶This ensures that newborns are screened for pediatric-onset conditions that are devastating but treatable when diagnosed early, providing opportunities for lifesaving medical interventions. The passage of this bill highlights the importance of bipartisan cooperation in advancing public health initiatives. To learn more visit: https://lnkd.in/e8eMstUk #newbornscreening #virginia #RUSP

Don’t miss this month's RDLA webinar! Stay ahead on key rare disease policy updates. We’ll cover the latest on budget reconciliation, priority review vouchers, the Accelerating Kids’ Access to Care Act, and the EXPERT Act. Register now at the link below: https://hubs.ly/Q03cm34q0

Learn how to share your story through art! 🎨 Join us for the Share Your Story Webinar, featuring inspiring Rare Artist Awardees and a special workshop with Stephanie Riordan on making postcards for your legislator. Don't miss this creative advocacy experience! #RareArtist #AdvocacyThroughArt Register at the link below: https://hubs.ly/Q03cyrKS0

Calling all rare disease advocates! Are you ready for some advocacy training? Level-up your skills with Rare Advocacy Learning! The focus of this seminar: Understanding the Rare Disease Community’s Imperatives. This free, six-week virtual seminar series is designed for experienced advocates! Hosted by RDLA, the program will equip you with the knowledge and tools to drive change. Deadline to apply: March 21, 2025 Don’t miss this opportunity—apply now! Head to the link below: https://hubs.li/Q03clPNT0

👉 Update: President Trump has signed a Continuing Resolution (CR) that will fund the government through September 30. However, the bill includes cuts to critical programs, including reductions to the Department of Defense’s Congressionally Directed Medical Research Program and key health agencies such as the NIH and HRSA. Unfortunately, the CR does not include language to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, which remains expired. This makes the push for reauthorization even more urgent. Both the House and Senate have introduced similar versions of the Give Kids a Chance Act, each proposing a five-year PRV extension. Now is the time for advocates to take action. Urge your members of Congress to prioritize this legislation and emphasize what’s at stake if the PRV Program is not swiftly reauthorized. Every day without action delays potential life-saving treatments for children with rare diseases. Let’s make our voices heard! Take Action on PRV: https://lnkd.in/gn3g-uX6 To learn more about how Congressional budget decisions shape rare disease funding visit: https://lnkd.in/eAMvzQaC

What is YOUR dream? #RAREis Scholarship Fund applications are now open! Thanks to the support of Amgen, The EveryLife Foundation for Rare Diseases established the fund to enrich the lives of adults living with rare diseases by providing support for their educational pursuits. This year, one-time awards of $5,000 scholarships will be provided to 104 rare disease recipients. Applications will close on April 28, 2025. Learn more and apply here: https://lnkd.in/eejYKsm

📣 Congress is juggling three separate but intertwined legislative processes related to funding government operations, each of which have direct implications for the rare disease community: ➡️ Continuing Resolution ➡️ Fiscal Year (FY) 2026 Appropriations Cycle ➡️ Budget Reconciliation The EveryLife Foundation helps explain what is happening, what is at stake, and what can be done to support the resources and services that drive our rare disease research, therapy development, and access to care. Learn more and take action here: https://lnkd.in/eAMvzQaC #policy #takeaction #raredisease