Rare Disease Week 2025

Why advocating for people with disabilities, rare diseases, and chronic illnesses is important to the LGS Community!

Every year, during the last week of February, advocates representing dozens of rare diseases, take time from their chaotic, medically complex lives and travel to Washington, D.C. to raise awareness of the needs of the rare disease community. And LGS proudly counts itself among that group.

With a new administration at the helm, the healthcare landscape is rapidly evolving. As we all know, LGS does not discriminate or care how you vote. Our families are in a constant state of crisis. We need research funding for cures, we need funds to provide the lifelong care that our loved ones require, we need access to expert healthcare providers, we need A LOT! And fortunately, we’re not afraid to ask for it.

So how does one ask for support? Very simply, you share your story. Gathered in D.C. last week were hundreds of individuals with hundreds of compelling stories. Our representatives want to hear those stories. How else will they know if the legislation that they’re proposing will have the right impact on their constituents?

Advocating can seem overwhelming to our caregivers. “I don’t know how”, “I don’t understand politics”, “I’m just one person. What could I possible do?”, “I don’t know where to begin”, “Others are already doing this, so I’m not needed”, and so on… But your voice IS needed. And it’s needed now, more than ever before.

How will all the potential changes affect the LGS community? We don’t know. That’s the real truth.

And that’s why Jamie, Sandy, Artika, Kurt, and I headed to D.C. for an event hosted by the Rare Disease Legislative Advocates. After a full-day conference outlining many of the needs of the rare disease community and specifically addressing four pieces of legislation, we the met with our state representatives to ask our members of Congress to support these ‘asks’.

The Proposed Asks

• Ensure Steady and Robust Federal Biomedical Research Funding & Public Health Agency Support – The removal of key experts at Health and Human Services Agencies, including NIH, FDA, and CDC paired with recent executive orders on federal funding and public health agencies will have a devastating and lasting impact on programs that are vital to thousands of rare disease communities, and to our nation as a whole.
• Reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program at FDA – To be eligible for a PRV, a treatment must obtain a Rare Pediatric Designation from the FDA, be eligible for priority review, and it must be the first approval for the drug’s active ingredient. The PRV program was last reauthorized in 2020 and expired in December 2024.
• Include Accelerating Kids’ Access to Care Act (AKACA) in the first available legislative package – In states where specialized care is not available, many children with complex medical needs often have to travel outside their home states for treatment. Programs like Medicaid and Children’s Health Insurance Program (CHIP) often limit kids’ ability to receive timely treatment outside their home state. This proposed legislation will ensure better, faster healthcare for these children.
• Ask Your Member of Congress to join the Rare Disease Congressional Caucus – This caucus works to raise awareness of rare diseases and the needs of the rare disease community.
• We could also include additional asks if they were relevant to the rare disease community. LGS Advocates were requesting that the representatives sign on to the National Plan for Epilepsy Act.

Our Advocates for LGS Team

• On a sad note, one of our most passionate advocates experienced an unfortunate rare disease roadblock that is all too common in the LGS community. New Hampshire advocate Kurt Oberhausen’s daughter Addison experienced a health crisis that landed her in the ICU. Kurt had to return home shortly after arriving in D.C. and couldn’t attend the Rare Disease Week activities. THIS is a rare disease experience at its worst, but Kurt is determined to continue sharing his family’s story. He will not be daunted because Addison’s story deserves to be told. Happily, she is now back home and will continue her LGS fight! Knowing that Addison was in the hospital, only fueled the rest of us to go out there and share our own LGS stories.
• Sandy Reynolds, from Pennsylvania, stated “On the Senate side, I had a short meet and greet with new-to-office Senator McCormick. I also was able to meet his staff members…He seemed interested and had his staff come back and ask specific questions and said they would all be sitting down for a meeting to discuss.” On the House side, Sandy met with a staff member for Representative Schmucker and shared “She was inquisitive and seemed interested in our daily challenges…Overall, I felt heard, and I hope that every person felt that they were making a difference.”
• Jamie Riley, a seasoned New Hampshire advocate, shared that she “had the extreme honor of personally meeting NH Senators Jeanne Shaheen and Maggie Hassan. They were receptive to hearing Andrew’s story and the importance of Kids Access to Care and the importance of being able to cross state lines for treatment. They also know the risks to Andrew and others with rare diseases on cutting Medicaid and Medicare.”
• As for my own meetings, they were with staff representing House Members Ro Khanna, Salud Carbajal, Jimmy Panetta, and Senator Adam Schiff in my home state of California. I felt heard, but I was also acutely aware that I was speaking to members of the minority party. However, I am hopeful that both parties will find a way to make our families needs a priority.

I think that our job now as a community is to stay in touch with our legislators. Know who represents you. You can find your representative here. If you would like more information about your representatives, check out the Legislative Scorecard on the EveryLife Foundation website. Consider respectfully letting them know how you feel about the proposed legislation.

In the coming months, we may need to reach out to certain members who may be considering supporting rare disease legislation that will impact your family. If so, I might reach out to you and ask if you can contact that person and share your story so they can hear the real-world implications of their decisions. As Sandy said, “The people that we meet daily don’t understand what our life is like, so the only way to explain is to talk about it. It’s not begging for attention. It’s making the world aware.”

And finally, please consider joining the Advocates for LGS team!

What Can We Do?

We stay informed, we share information with our community, we participate when we can (LGS can sometimes interfere with that and we understand), we respond to Calls to Action, and we teach you how to be an advocate. That last one is very important. You don’t need to know how to do this. You just need to know what you already know, YOUR LGS STORY!!

We know that this can be overwhelming. We’re here to help answer questions, share information and resources, motivate you to ACT and be a voice for the LGS Community.

-Written by; Jennifer Griffin, LGS Foundation Director of Family Support 

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Updated 03/06/25 (AM)