EveryLife Foundation for Rare Diseases

04/23/2026

📣 Last call! Applications for the Scholarship Fund close on Tuesday, April 28, at 2 PM Eastern.

58 individuals in the rare disease community will receive $5,000 scholarships to support their education this fall. Don’t miss this opportunity to take the next step in your education and career.

Apply now at https://hubs.li/Q04dbDBp0.

04/23/2026

‼️ Today, Congress is taking the first step on appropriations to fund the Food and Drug Administration’s FY27 operations, including the critical work the FDA does to evaluate therapies for rare diseases. In advance of the markup, the EveryLife Foundation submitted a statement to the subcommittee, urging lawmakers to support the inclusion of funding for the Rare Disease Innovation Hub, an ask first made by the hundreds of advocates who joined us in Washington, DC for Rare Disease Week 2026.

The rare disease community is requesting that the Hub receive $5 million in funding to carry out its agenda, which includes hosting additional RISE Workshops, creating opportunities for rare disease patient organizations and experts to inform the therapy development and evaluation process, and streamlining navigation of the FDA’s rare disease resources. Dedicating funds to the RDIH ensures that the Hub can continue to grow its role in improving coordination across the FDA, leading to more consistent approaches to rare disease therapy development.

This request is supported by more than 25 Congressional members who sent a letter to the subcommittee in March, and we look forward to continuing to work with Congress to ensure that the Rare Disease Innovation Hub is resourced and can work to its fullest potential.

Share your support for rare disease funding at the FDA ➡️ https://everylifefoundation.quorum.us/campaign/157296/

Read the full statement on our website➡️ https://everylifefoundation.org/the-everylife-foundation-submitted-a-statement-to-congress-urging-lawmakers-to-support-the-inclusion-of-funding-for-the-rare-disease-innovation-hub/

04/16/2026

From June 9-11 in Boston, MA, the World Orphan Drug Congress USA will once again bring together pharmaceutical companies, government officials, patient advocates, and other crucial stakeholders who contribute to developing and improving access to life-saving therapies for rare disease patients. With over 280 speakers and 2,000 attendees, this event is an opportunity to develop innovative solutions to the challenges that orphan drug development poses.

Our Chief Mission Officer, Annie Kennedy, will speak on the first day of the event at 9:20 am during the keynote titled, “Building a Patient-Centered Policy Framework for Rare Disease Drug Development.” The keynote will focus on how policymakers, regulators, and industry can collaborate to create strategies that incorporate patient insights into all stages of drug development.

You can join Annie and the EveryLife Foundation at this event for free with a guest pass. Click the following link to access the guest pass application form to secure a spot: https://secure.terrapinn.com/V5/sponsor-guest/11031/a0AN200000inZjFMAU?utm_source=partners&utm_medium=everylifefoundation&utm_campaign=part&trc=part

You can learn more about this year’s World Orphan Drug Congress USA here: https://www.terrapinn.com/conference/world-orphan-drug-congress-usa/index.stm?utm_source=partners&utm_medium=everylifefoundation&utm_campaign=part&trc=part

04/10/2026

This week, the EveryLife Foundation for Rare Diseases was proud to co-host the 2026 in Boston, MA, in partnership with Ultragenyx, bringing together advocates, families, and leaders across the rare disease community for three days of immersive learning and connection.

Through expert-led discussions and collaborative conversations, attendees gained practical guidance, resources, and strategies to help move rare disease drug development efforts forward. EveryLife Foundation CEO, Michael Pearlmutter, spoke during yesterday's event.

Each Rare Bootcamp is a reminder of what’s at stake for rare disease families and shows the power of collaboration in pushing the boundaries of what’s possible. Thank you to our sponsors, Ultragenyx, Goodwin, Chiesi Global Rare Diseases, Rentschler Biopharma, Alexion Pharmaceuticals, Alnylam, BioMarin, BridgeBio, GeneDx, Dash Bio, iXCells Biotechnologies, Morgan Lewis, Neucyte, and Global Genes for making this event possible!

04/08/2026

The 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 https://hubs.li/Q049_wpm0

04/06/2026

‼️ 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 ➡️ https://everylifefoundation.org/the-presidents-budget-proposal-what-does-it-mean-for-our-rare-disease-community/

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.

04/02/2026

‼️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 ➡️ https://everylifefoundation.quorum.us/campaign/159549/

Read more about the bill ➡️ https://calmatters.digitaldemocracy.org/bills/ca_202520260ab1798

We look forward to following this bill's progress as it advances through the California legislature.

EveryLife Foundation for Rare Diseases

04/01/2026

📅 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 Duke-Margolis 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 ➡️

03/27/2026

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: https://hubs.li/Q048FqCl0

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: https://hubs.li/Q048FzV30

03/27/2026

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:
Alexion, Sanofi, Amgen, Merck, Takeda, argenx, Travere Therapeutics, Biohaven, Harmony Biosciences, Beam Therapeutics, Biogen, CSL, Genentech, GSK, Pfizer, UCB, Acadia, Amicus Therapeutics, BioCryst, BioMarin, Biotechnology Innovation Organization, Boehringer Ingelheim, Chiesi Global Rare Diseases, Faegre Drinker, Ionis, Jazz Pharmaceuticals, Lundbeck, Neurocrine Biosciences, Otsuka, PTC Therapeutics, Recordati Rare Diseases, Soleno Therapeutics, Scholar Rock, Stoke Therapeutics, Sumitomo Pharma America, Ultragenyx.

Read the full Recap Here: https://everylifefoundation.org/everylife-foundation-for-rare-diseases-emboldens-advocates-and-urges-congress-to-prioritize-patients-during-15th-annual-rare-disease-week-on-capitol-hill/

03/26/2026

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: https://hubs.li/Q048t9hH0

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: https://hubs.li/Q048v5bb0

03/20/2026

📅 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 ➡️ https://everylifefoundation.org/event/north-carolina-state-advocacy-day/