Blog

2026 Rare Diseases Symposium: Bringing Histio to the Table
By Melinda Atnip – Program and Outreach Coordinator

This year marked my third time attending the Rare Diseases Symposium, co-hosted by CHOC Research Institute (now part of Rady Children’s Health) and University of California, Irvine at the Beckman Center of the National Academies of Sciences & Engineering. Over the years, I’ve watched this event grow—from primarily clinical research to including more patient and family voices.

This year, histio ambassador Dena and I were thrilled to have an awareness table for histiocytosis, representing the Histiocytosis Association. Out of all the groups attending, there were only eight other rare disease organizations—we were very much in the minority. That made our presence even more important.

We set up an eye-catching display filled with educational materials and resources for physicians, including: CME opportunities on recognizing and treating HLH, Impact Cards, Histiocyte Society info, disease-specific materials, and even a few “Histio Sucks” lollipops and gummy bears. I made organized takeaway bags so physicians could easily access the information without fuss.

Most of the day was spent talking directly with physicians, researchers, lab scientists, and department leaders from institutions like UCLA, Stanford University, UCI, and CHOC. Gene therapy and genomic sequencing were hot topics—cutting-edge areas that could directly impact our community.

One of the highlights was seeing Dena share her story. A physician treating a histiocytosis patient even sat with her for over an hour to learn more. Moments like that are why we keep showing up—connecting families, knowledge, and research in meaningful ways.

Looking back, it’s amazing to see the growth: the first year our physicians presented posters, the second year I submitted a poster on care gaps identified through our histio physician directory, and now a full table to share our community’s experiences. The symposium continues to expand patient involvement, reminding everyone that behind every discovery are real families waiting for answers, hope, and healing.

Even in a room where we were one of the few rare disease groups, histio had a strong, visible presence. In my own backyard, we were able to connect directly with leading physicians and researchers, share critical resources, and ensure that histiocytosis is part of the conversation—one meaningful conversation at a time.

Another whirlwind Rare Disease Day activity! After a quick outfit change, I headed to Laguna Beach to support our new coalition, partner, Raregivers. They had a fundraiser and the screening of The Greatest Showman, featuring a special appearance by actor Sam Humphrey—a beautiful way to end a wonderful Rare Disease Day.

Dena

Rare Disease Week on Capitol Hill

By Nate S. and Molly Zangrilli

Histio Youth Ambassador Nate

Rare Disease Week is an incredible opportunity to advocate for crucial change regarding rare diseases as well as connecting with others within the rare disease community. This was my second year participating, and I am continuing to learn what it means to be an effective advocate for rare disease legislation and how to best communicate the needs of this community to policymakers.

This year, I had the opportunity to meet with several Maryland representatives as well as Washington, D.C.’s non-voting representative, Eleanor Holmes Norton. Similar to last year, my advocacy groups were relatively small, which allowed most people to share their personal experiences and present their “asks”. In all four meetings, I was able to tell my story and advocate specifically on strengthening the development of treatments for rare diseases. In particular, I advocated for congressional support and appropriations for the FDA’s Rare Disease Innovation Hub.

The Rare Disease Innovation Hub was created within the FDA to improve coordination across the agency’s many offices that work on rare disease research, regulation, and drug development, like the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), with limited centralized coordination. The Innovation Hub is intended to address this by creating a structured framework for collaboration among scientists, regulatory experts, and patient engagement teams. By improving internal communication and aligning regulatory expertise, the Hub aims to accelerate the development and review of therapies for rare diseases. With more than 7,000 known rare diseases and only a small fraction having approved treatments, strengthening this type of infrastructure within the FDA has the potential to significantly improve the pace of rare disease treatment development.

Rare diseases are also a bipartisan issue, and my meetings on Capitol Hill reflected that. Each of the offices I spoke with showed genuine interest in the issues we raised and seemed receptive to supporting our requests. On Thursday, I met in person with three Maryland congressional offices, and on Friday I met with Representative Eleanor Holmes Norton over Zoom. Each meeting was productive, and nearly everyone in our group had the opportunity to speak.

One thing I have learned through these experiences is the importance of approaching meetings with an open mind. Even when speaking with the office of a representative whose positions may differ on other policy issues, there is often still meaningful common ground when it comes to rare diseases. In fact, the meeting I initially expected to be the least productive ended up being one of the most engaging and impactful conversations I had.

Seeing so many people from across the country come together in one place to work toward improving the lives of those affected by rare diseases was incredibly inspiring. The sense of shared purpose and community made the experience especially meaningful, and it reinforced how powerful patient advocacy can be. I am grateful to have participated again this year and am already looking forward to attending Rare Disease Week next year!

Histio Ambassador Molly

I had the privilege of representing the Histiocytosis Association at the EveryLife Foundation’s Rare Disease Week in Washington, D.C. It was an energizing few days and included advocates, caregivers, clinicians, and leaders working to advance the causes critical for the rare disease community.

Day One: Legislative Momentum and Community Stories: The first day of the conference featured key legislative updates and a celebration of the reauthorization of the Rare Pediatric Disease Priority Review Voucher Program. The EveryLife Foundation has spent more than a year championing this effort, and it was inspiring to witness such a meaningful win for families affected by rare diseases.

We also heard several powerful patient and caregiver stories. One especially moving moment came from the Barth Syndrome Foundation, whose Executive Director shared their long advocacy journey toward accelerated approval of elamipretide, the first treatment for Barth syndrome, a rare genetic mitochondrial disease. Approved in September 2025, the journey was highlighted through a short documentary that they shared with us. Many foundation members attended the conference and received heartfelt applause when recognized.

I also got to meet fellow HA member and Youth Ambassador Nate S. on Day 1!  Melinda introduced us over email and we texted ahead of time.  By a crazy coincidence, Nate happened to be sitting at the same table as a friend of mine from 2025 who had saved me a seat!  Small world!

Day Two: Advocacy on Capitol Hill The second day focused on meetings with legislators. Pennsylvania had an impressive presence this year, with 55 rare disease advocates—the largest state delegation at the conference. I met with staff from the offices of U.S. Representative Chrissy Houlahan, U.S. Senator Dave McCormick, and U.S. Representative Lloyd Smucker. Each conversation felt productive and I was heartened to see the genuine empathy and concern the staffers exhibited.

My advocacy centered on the Genomic Answers for Children’s Health Act, which aims to improve outcomes for children with rare, undiagnosed diseases by requiring Medicaid coverage for whole genome and whole exome sequencing. As a member of the alliance supporting this bill, I was grateful to thank Rep. Houlahan for being an early co‑sponsor. I also encouraged her office to join the Rare Disease Congressional Caucus to deepen engagement with the challenges our community faces.

I had a great time at my second Rare Disease Week and I cannot wait to return next year!