EveryLife Foundation

The #RAREis Scholarship supports individuals with rare diseases in building their dreams. Beyond financial support, recipients are invited to participate in a mentorship program that connects them with peers who share similar experiences or areas of study. Apply by April 28 at 2 PM ET at hubs.li/Q049_HtF0

‼️ Last Friday, April 2, the White House released the President’s proposed Budget for Fiscal Year 2027. It proposes $15.8 Billion in reductions to Health and Human Services (HHS), including significant funding cuts to key agencies for the rare disease community, and an increase to the Food and Drug Administration (FDA). This budget is by no means final. Congress views the President's budget as a starting point for the budget process, but often chooses to draft based on Congressional priorities instead. While there is still much we will learn and distill over the coming days and weeks as negotiations on the Hill evolve, we wanted to take this opportunity to provide an overview of the significance of the release of the President’s Budget Proposal and the process that comes next. To read the full post, please visit our website ➡️ everylifefoundation.org/the-presidents… Our EveryLife Foundation team is here with you. We will keep you updated as new developments are shared, hearings are posted, and key opportunities arise to ensure that the funding priorities of our community are heard on the Hill.

‼️Attention California residents‼️ The California General Assembly has introduced A.B. 1798, a critical bill that protects patients from genetic discrimination and ensures continued participation in lifesaving research. Members of the Assembly Committee on Insurance have the opportunity right now to support this important legislation. Your story matters! Share why genetic nondiscrimination matters to you and how it impacts your life. Urge committee members to support this bill by filling out our action alert ➡️ everylifefoundation.quorum.us/campaign/15954… Read more about the bill ➡️ calmatters.digitaldemocracy.org/.../ca... We look forward to following this bill's progress as it advances through the California legislature.

📅 The RISE Workshop 3 convened on March 30, 2026, to discuss optimizing data sharing for therapy development and the collaboration, infrastructure, and education opportunities to promote data sharing as a consistent reality in the rare disease space. The EveryLife Foundation was delighted to support Duke Margolis Institute for Health Policy and the FDA Rare Disease Innovation Hub during the planning of this workshop, as the meeting topic was based on topic submissions from the community, including our July submission to the Federal Register. Annie Kennedy, Chief Mission Officer, was invited to provide opening remarks to help set the tone for the day's discussions. These hybrid RISE workshops are co-convened by the @DukeMargolis Institute for Health Policy and the U.S. Food and Drug Administration (@FDA) Rare Disease Innovation Hub under a cooperative agreement with the FDA. RISE workshops bring together innovators in drug development, rare disease research, patient advocacy, and regulatory science to discuss challenges in the development of medical products for rare diseases that are common to multiple rare diseases or a class of diseases and for which evolving science offers innovative solutions. Thank you to our partners at the Rare Disease Innovation Hub and at Duke-Margolis for convening this workshop and facilitating important conversations around this topic. To learn more about this week's workshop, click here ➡️ everylifefoundation.org/rise-workshop-…

Ready to share your story and take part in rare disease advocacy? Join the EveryLife Foundation for Rare Diseases for a virtual session, “How to Get Involved,” on Thursday, April 9, at 6 PM ET. You’ll hear about our programs, events, and the many ways to engage in advocacy through the Foundation. Register now at: hubs.li/Q048FBqG0 Can’t attend in April? These sessions will be offered every other month on the second Thursday at 6 PM ET. Want to start connecting now? Sign up for our newsletter so you never miss an opportunity: hubs.li/Q048FkYR0

From February 24-26, 2026, the EveryLife Foundation for Rare Diseases convened hundreds of patients, caregivers, advocates, researchers, and policymakers from 49 states, the District of Columbia, and Puerto Rico for the 15th annual Rare Disease Week on Capitol Hill. Rare disease community members spoke to their Members of Congress to advance bipartisan policy solutions aimed at accelerating diagnoses, supporting families, and strengthening rare disease research and regulatory innovation. Thank you to all our attendees and staff for making this incredible event possible. When passionate individuals come together, powerful change happens. Thank you to all our sponsors for their support: @AlexionPharma,@sanofi, @Amgen, @Merck, @TakedaPharma, @argenxglobal, @TravereRare, @biohaven, @HarmonyBio, @BeamTx,@biogen, @CSLBehring, @genentech, @GSK, @pfizer, @ucb_news, @AcadiaPharma, @amicusrx1, @BioCrystPharma, @BioMarin, @IAmBiotech, @Boehringer, @ChiesiGRD, @FaegreDrinker, @ionispharma, @JazzPharma, @LundbeckUS, @neurocrine, @OtsukaUS, @PTCBio, Recordati Rare Diseases, @ScholarRock, Soleno Therapeutics, @StokeTx, Sumitomo Pharma, @ultragenyx. Read the full #RareDC2026 Recap Here: everylifefoundation.org/everylife-foun…

During Rare Disease Week, advocates urged Congress to pass the Genomic Answer for Children’s Act (H.R. 7118) to expand access to life-changing genomic testing for children covered by Medicaid. Children with rare diseases often face years-long delays in getting an accurate diagnosis, making timely access to genomic testing critical. However, not all state Medicaid programs cover these necessary tests, leading to unnecessary diagnostic and treatment delays. The Genomic Answers for Children's Health Act makes clear that whole-genome sequencing and whole-exome sequencing are covered Medicaid services, promotes awareness among stakeholders, and requires new reports to Congress on barriers to care. This legislation will help shorten the diagnostic odyssey and connect children and families with the answers and care they deserve. Encourage your Representative to support the bill here and check back soon for opportunities to advocate to your Senators once a Senate version is introduced: hubs.li/Q048v3b20 Today at 2 pm ET, Victoria Gemme, Director at Leavitt Partners, will discuss the Genomics Answers for Children’s Health Act at the RDLA Monthly Webinar. Register for the webinar here: hubs.li/Q048v3Bp0

📅 Join us for North Carolina State Advocacy Day! If you're a North Carolina resident, join us on May 18 at the City of Raleigh Museum in the Women’s Club Classroom from 1 PM – 4 PM EST for the legislative conference. Hear from local organizations, network with advocates from your state, and learn more about rare disease legislation. On May 19, meet us at the Capitol building for breakout groups to speak with your state legislators about the policy priorities of the rare disease community. Registration closes Friday, April 24. The deadline for travel reimbursement is April 3. To learn more and register for North Carolina State Advocacy Day ➡️ everylifefoundation.org/event/north-ca…

This past Sunday, the EveryLife Foundation proudly celebrated our inaugural team at the United Airlines NYC Half Marathon and what an incredible success it was! Six dedicated runners took to the streets of New York City, each mile powered by purpose, determination, and a deep commitment to the rare disease community. Watching them cross the finish line was more than a race moment, it was a powerful reminder of what can happen when advocacy, community, and heart come together. To our six runners: THANK YOU… for your training, fundraising, dedication, and for choosing to run in support of individuals and families navigating rare diseases. Your efforts help raise awareness, strengthen our community, and advance the mission we share. Katie Barrett-Stevens, Katie Kundrat, Julie Evarts, Heli Salazar, Donta Gonzalez, and Kathleen Smart. We are also deeply grateful to this community: our supporters, advocates, donors, and friends, who rallied behind this team and helped make this milestone possible. This was our first team at the United Airlines NYC Half Marathon, and it marks the beginning of something truly meaningful. What started this year has laid a strong foundation for future growth and impact, setting the stage for what we hope will become a lasting tradition of engagement, community, and support for the rare disease community. You can still support this amazing team! Every contribution helps advance research, advocacy, and support for individuals and families affected by rare diseases. Join us in celebrating their achievement and continuing the momentum: hubs.li/Q047qh7r0

The #RAREis Scholarship Fund is approaching its 500th recipient since launching in 2020. Could it be you? Applications are now open for the #RAREis Scholarship Fund. Thanks to the support of @Amgen's RAREis program, the EveryLife Foundation for Rare Diseases will award 58 scholarships of $5,000 to individuals in the rare disease community pursuing higher education in Fall 2026. Learn more and apply at hubs.li/Q04758mn0. Application closes April 28 at 2 PM Eastern.

The NYC Half is almost here! This weekend the EveryLife Foundation Run Team will run on behalf of the 30 million Americans living with rare diseases. Help us cheer them on and support their mission. 🔗 Make your donation today! hubs.li/Q046qvKv0

The EveryLife Foundation urges Congress to support funding and report language in FY 2027 for the Rare Disease Innovation Hub. The Hub facilitates collaboration between the FDA’s Center for Biologic Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) to improve outcomes for patients living with rare diseases. Our Chief Mission Officer, Annie Kennedy, urged Congress to resource the Hub in her witness testimony at the Senate Special Committee on Aging last Thursday during Rare Disease Week 2026. Currently, only 5% of the more than 10,000 rare diseases have FDA-approved treatments. The approval process for rare disease drugs requires special knowledge and skills across FDA departments. Without clear collaboration, it is challenging to compete the approval process in a timeframe that meets rare disease patients’ needs. The Hub streamlines collaboration between departments to ensure the agency can meet patients' needs. With proper funding and clear guidelines, the Rare Disease Innovation Hub can improve the FDA’s rare disease drug approval process and transform the lives of patients nationwide. Encourage your Member of Congress to support funding for the Rare Disease Innovation Hub here: hubs.li/Q045KrXS0 To watch the full Senate Special Committee on Aging hearing here: hubs.li/Q045KrNm0

We’re heading into the final stretch before the New York City Half Marathon! Six dedicated runners are taking on the NYC Half on behalf of the EveryLife Foundation for Rare Diseases, running in honor of the 30 million Americans living with a rare disease and the families, caregivers, and advocates who stand beside them every day. Because of this incredible community, we’ve already reached 84% of our fundraising goal. With race day quickly approaching, we’re inviting you to help us cross the finish line together. ✨ Support their journey with a donation here: hubs.li/Q045C_6X0 Every gift fuels our mission, advancing evidence-based policy, accelerating science-driven research, and strengthening the rare disease community so that no one has to navigate their journey alone. Thank you for cheering on our runners and standing with the rare disease community every step of the way.

During Rare Disease Week 2026, Congressman Josh Gottheimer announced that the bipartisan Problem Solvers Caucus endorsed Cameron’s Law! This legislation fully restores the Orphan Drug Tax Credit and supports investment in rare disease treatments. The EveryLife Foundation is grateful to the Problem Solvers Caucus for recognizing the importance of Cameron’s Law, and we encourage Congress to move the bill forward this year. #RareDC2026 Read the full announcement here: gottheimer.house.gov/posts/release-…

LISTEN: Annie Kennedy from @EveryLifeOrg shares the heartbreaking reality of how bureaucracy blocks lifesaving care those with rare diseases. It’s time to cut through the red tape & deliver SAFE RESULTS so more Americans with rare diseases can grow up & grow old.

What a way to spend the final days of Rare Disease Week 2026! Despite a little drizzle and some gray clouds, hundreds of rare disease advocates participated in more than 300 Hill visits on Thursday to share their stories and advocate for rare disease policies. Yesterday started with our community in the Senate spotlight. The EveryLife Foundation for Rare Diseases was honored to help represent the community by participating in the @SenateAging hearing on the effects of FDA delays on rare disease therapy development. Annie Kennedy, our Chief Mission Officer, joined other expert witnesses, Dr. Jeremy Schmahmann, Director of Massachusetts General Hospital Ataxia Center; Bradley Campbell, President & CEO of Amicus Therapeutics; and Dr. Cara O'Neill, Chief Science Officer and Co-Founder of the @CureSanfilippoF, to give expert testimony. We would like to extend our deepest gratitude to Chairman @SenRickScott, Ranking Member @SenGillibrand, @SenJohnson, @SenMcCormickPA, @Sen_Alsobrooks, and @SenatorAndyKim for the important dialogue in this hearing. The expert witnesses focused on issues regarding the utilization of existing regulatory tools, such as the accelerated approval pathway and advisory committees; the need for consistency and transparency in the drug review process; ensuring ethical practices in rare disease clinical trials that do not result in unnecessary disease progression; and increasing domestic manufacturing transparency and speed to fulfill the United States' innovation capabilities. Our message, our stories, and our urgency were heard. Conversations with the Senate Special Committee on Aging will continue in the coming days. We will keep you updated on any future Committee interactions. For more information, visit our website: everylifefoundation.org/rare-disease-w… There is still time to share your story about how regulatory delays and the lack of treatment options have affected your life before the Committee’s deadline. Share your story before March 3: everylifefoundation.quorum.us/campaign/15558…

On Monday, the @US_FDA released more details about the Plausible Mechanism Framework. The Plausible Mechanism Framework is a set of recommendations to help those developing individualized therapies generate sufficient evidence that they are safe and effective, and that they can be manufactured appropriately. The EveryLife Foundation team will closely review the draft Guidance, alongside the Community Congress Regulatory Working Group in the coming weeks and together, we will continue to press for policy solutions that acknowledge the differences in how rare disease products must be developed and evaluated. Today’s release of the Plausible Mechanism Framework Draft Guidance was a meaningful step in that direction. To learn more about yesterday's event at the @HHSGov visit: everylifefoundation.org/fda-takes-impo…

UPDATE: the first session now features Mark McClellan with Jay Bhattacharya, Director at the National Institutes of Health and acting Director at the Centers for Disease Control and Prevention. Register here: washingtonpost.com/washington-pos…

Happening soon! Tomorrow at 3 pm ET, The Washington Post is hosting conversations about the next frontiers of rare disease research. Representative Doris Matsui, co-chair of the Rare Disease Congressional Caucus, joins Mark McClellan, the Duke-Margolis Institute for Health Policy, for one conversation. Then, our Chief Mission Officer, Annie Kennedy, joins Cate McCanless, Chief Corporate Affairs Officer at Harmony Biosciences, for a discussion focusing on the role of patients in rare disease policy in addressing their urgent needs. Register to watch here: hubs.li/Q044fRt_0