EveryLife Foundation

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

‼️Our Chief Mission Officer, Annie Kennedy, will be testifying at the Senate Special Committee on Aging's hearing that looks to examine the FDA's role in the rare disease community. The hearing will be held on February 26 at 9:30 am in the Hart Senate Office Building, room SH-216. If you are attending Rare Disease Week, join us for a breakfast and a meet and greet at 8:30 am in the Dirksen Senate Office Building, Room G-11, before or between your Hill meetings. If you are not attending Rare Disease Week and would still like to tune in, you can watch the livestream here: aging.senate.gov/hearings In recent months, several difficult regulatory decisions have affected clinical trials and review timelines for products with direct implications for the rare disease patient community. Remember, your story can help inform what happens next! The Aging Committee is collecting stories about what rare disease research and treatments mean to patients, highlighting the real-world impact of regulatory delays on individual rare disease communities. Share your story here ➡️ everylifefoundation.quorum.us/campaign/15558…

‼️ The Senate Special Committee on Aging has announced it will hold a hearing to examine the FDA's role in the rare disease community. The hearing will be held on February 26 at 9:30 am in the Hart Senate Office Building, room SH-216. If you are attending Rare Disease Week, join us for a breakfast and a meet and greet at 8:30am in Dirksen Senate Office Building, Room G-11 before or between your Hill meetings. We will be watching a livestream of the hearing from this room as well, if the main room reaches capacity. If you are not attending Rare Disease Week, you can watch the livestream here: aging.senate.gov/hearings ❓ Why this Hearing is Important In recent months, several difficult regulatory decisions have affected clinical trials and review timelines for products with direct implications on the rare disease patient community (for example, complete response letters, refusals to file, and incomplete response letters). If you are a member of a patient community affected by these decisions, we want to hear from you. To help inform the hearing, the Aging Committee is collecting stories about what rare disease research and treatments mean to patients, highlighting the real-world impact of regulatory delays on individual rare disease communities. Share your story here ➡️ everylifefoundation.quorum.us/campaign/15558…

Why is Katie Kundrat running: "Katie and Kathleen are running together as a fundraising team, combining their efforts to support the rare disease community and amplify their collective impact. I run because every day I work with children and families navigating rare neuromuscular conditions, and I see how much strength and perseverance their journeys require. The families and patients endless dedication to their care and improving their quality of life, inspires me to move and run for this cause.  As a physical therapist on the neuromuscular clinical team at Children’s National, I support patients as they work toward mobility, strength, and maintaining quality of life  and those experiences stay with me long after the clinic day ends. Running with the EveryLife Foundation feels like another way for me to stand alongside the patients and families I care for. This race is about showing up, raising awareness, and helping advance a future where individuals affected by rare disease have the support, research, and resources they deserve. I am proud to represent a foundation that embodies the same values my patients exhibit every day." To help Katie reach her goal, click here: hubs.li/Q043cvzp0 To learn more about the rest of the EveryLife Foundation for Rare Diseases Run Team, click here: hubs.li/Q043cvmk0 #RaceforRare

Why is Donta running: “I wanted to run the NYC Half with the EveryLife Foundation because their mission aligns deeply with my commitment to advocacy, equity, and community-driven impact. Rare disease patients and families too often navigate fragmented systems without adequate support or representation, and the EveryLife Foundation works tirelessly to change that by amplifying patient voices and advancing meaningful policy solutions. Running this race is my way of honoring the resilience of the rare disease community and helping ensure that lived experience is at the center of research, policy, and care. Every mile represents progress toward a future where no rare disease patient is left behind.” To help Donta reach his goal, click here: hubs.li/Q0420fZ50 To learn more about the rest of the EveryLife Foundation for Rare Diseases Run Team, click here: hubs.li/Q0420gpQ0 #RaceforRare

As the rare disease community celebrates the reauthorization of the PRV Program, we want to take a moment to thank the leaders of the Give Kids a Chance Act for their unwavering support. Thank you to Representatives @RepMcCaul, @RepDebDingell, @RepGusBilirakis, @DorisMatsui, @RepKimSchrier, @RepHarshbarger, @USRepKCastor, @MikeKellyPA, @RepDanCrenshaw, @RepLoriTrahan, and @TXRandy14, as well as Senators @SenMullin and @SenatorBennet. The rare disease community is grateful for your advocacy!

‼️Breaking News: Rare Pediatric Disease PRV Program Reauthorized by Congress! After a two-year campaign to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, the rare disease community’s relentless advocacy has paid off. Congress has passed the Labor, HHS, and Related Agencies Appropriations bill, effectively reauthorizing the PRV Program for five years while also funding a number of other critical healthcare agencies. We applaud the reauthorization of the PRV Program and renewed investments in critical health research and public health programs. Thank you to the congressional champions who have partnered with our rare disease community to secure these advances. While significant work remains to enable all those living with rare diseases to thrive, today’s progress will accelerate innovation, expand access to life-changing therapies, and offer renewed hope to children and families whose futures once seemed beyond reach. To learn more about the full healthcare package, please visit our website: everylifefoundation.org/congress-passe…

Hiring Now! The EveryLife Foundation for Rare Diseases is hiring for a Director of Events. We are looking for an experienced leader in event management, preferably in a non-profit, healthcare, or advocacy setting. The EveryLife Foundation hosts in-person, hybrid, and virtual events that empower rare disease advocates to make their voice heard with policymakers. The Director of Events is the bridge between complex policy work and the people it impacts. Interested applicants can use the following link to learn more about the position and apply: hubs.li/Q041_NPf0

Why is Kathleen running: “I run because every day I work alongside children and families navigating rare neuromuscular conditions, and I see how much strength it takes to live with uncertainty. As a Neuromuscular Clinic Coordinator at Children’s National, I’ve had the privilege of supporting families through some of their most challenging moments — and those experiences stay with me. Running with the EveryLife Foundation feels like a natural extension of the advocacy I care so deeply about. This race is my way of showing up for the rare disease community, helping raise awareness, and supporting efforts that bring families closer to answers, resources, and hope. Every mile is for the patients and families who inspire me every single day.” To help Kathleen reach her goal, click here: hubs.li/Q041jHF50 To learn more about the rest of the EveryLife Foundation for Rare Diseases Run Team, click here: hubs.li/Q041jH1P0 #RaceforRare

‼️Update: Senate Advances Healthcare Funding and Rare Disease Priorities Today, the Senate passed an amended government funding package that includes five FY26 appropriations bills and a set of important healthcare policies. The legislation would increase NIH funding for rare disease research, reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program for five years, and make it easier for kids with rare diseases who have Medicaid to access care across state lines. We are grateful to Senate leaders for advancing these time-sensitive policies and sustaining funding for key healthcare agencies through September 30, 2026, especially given the dynamic legislative landscape. Because the Senate made changes to the legislation the House passed, the process is not yet complete. ➡️What’s next: The House is expected to consider the amended package when it returns to Washington on Monday. If approved, the bill would move to the President’s desk for signature. In the meantime, a partial government shutdown is set to begin January 31 and will continue until final legislation is enacted. We will continue to monitor developments and share updates on potential impacts to the rare disease community.