Advocating for YOU: Rare Disease Week on Capitol Hill

Advocating for YOU: Rare Disease Week on Capitol Hill

Categories: Blog, Family Empowerment, Homepage, News

IRSF works tirelessly to advocate for you and your loved one with Rett syndrome. Our work takes many forms, our persistence is often unseen, and our successes are the result of countless hours spent meeting with, raising awareness, and educating key decision-makers on the needs of our Rett community.

Last month, our advocacy efforts were at the forefront as we spent the week raising awareness of Rett syndrome in Washington, D.C. for Rare Disease Week. Below, IRSF’s Government Relations Manager, Katie Busch, shares more about how critical these efforts are to bringing home treatments and cures, getting us that much closer to a world without Rett syndrome.

Katie and Ellie who has Rett syndrome.

As the longtime para and caregiver of a young woman with Rett syndrome, I’ve seen firsthand the strength it takes to fight every day to better the life of a loved one with Rett. I am so proud to be able to continue that fight with and for every family in my new role as IRSF’s Government Relations Manager.

Last week, I had the honor of joining Melissa Kennedy, IRSF CEO, in Washington, D.C. to represent the Rett community at this year’s Rare Disease Week held by the EveryLife Foundation.

Our first day on the Hill was spent in conference with over 800 fellow advocates. I listened to the policy priorities, met incredibly inspiring people, and heard amazing stories from presenters including Melissa who spoke on the impact a priority review voucher had on bringing DAYBUE (trofinetide) to our loved ones with Rett. I quickly realized that the hard work was just beginning. We were about to be set loose on the Hill to meet with our respective Representatives and Senators.

Armed with the policy priorities (and an abundance of hope and enthusiasm) my fellow Missouri advocates and I headed to House and Senate office buildings.  In the meetings with the Congressmember’s staffers, we shared stories of what living with a rare disease is like and how policies affect those living with a rare disease. We were making a difference and being heard.

IRSF CEO Melissa Kennedy speaks to over 800 rare disease advocates.

We met with:
Sen. Josh Hawley (R-MO)  |  Sen. Eric Schmitt (R-MO)  |  Sen. Van Hollen (D-MD)  |  Sen. Sherrod Brown (D-OH)  |  Sen. JD Vance (R-OH)  |  Rep. Abigail Spanberger (D-VA-07)  |  Rep. Greg Landsman (D-OH-01)  |  Rep. Max Miller (R-OH-07)  |  Rep. Ann Wagner (R-MO-02)  |  Rep. Cori Bush (D-MO-01)  |  The White House Office of Science and Technology Policy Health Outcomes Division

On my last day on the Hill, Melissa and I dedicated time to advocating for Rett syndrome research funding. We met with Rep. Abigail Spanberger’s office to thank her for her continued support for Rett research funding through the U.S. Department of Defense Congressionally Directed Medical Research Program. We were also joined by Steven Marconi (RTT Dad) in a meeting with his Maryland Senator to share what it’s like to live with Rett.

As I reflect on all the meetings and events one thing stands out in my mind: the stories. The struggles and triumphs that all the families shared. The similarities in the challenges and wins experienced by everyone whose life is impacted by a rare disease. Our Rett community is small and mighty, but we are also a part of something much larger. A united front.

Melissa Kennedy, Katie Busch, and Rett dad, Steven Marconi

Taking off on my return flight, I watched the lights of D.C. fade and disappear, but the excitement I felt upon arrival was replaced with a sense of motivation to continue advocating for you. I’m inspired by the rare disease community and our Rett community, who shine clear and bright in their mission, championing their cause to improve the world. And I’m personally thrilled to continue my advocacy for Rett research funding.


This was an impactful week for Rett syndrome, but the fight is not over. We promise to continue to advocate for:

  • The federal funding needed to support researchers broadening our understanding of Rett syndrome and translating their findings to real-world treatments.
  • Industry partners and biopharmaceutical companies to invest their resources in completing successful clinical trials and building the infrastructure needed to deliver treatments to patients all over the world.
  • Insurance companies and state Medicaid programs to keep current and future treatments affordable and accessible so our entire community can benefit.


Secure Federal Research Funding
Every year we need you to help us advocate for Rett to be included as a topic eligible for funding through the U.S. DOD funding bill. While we eagerly anticipate Congress finalizing the FY24 funding bill, the fight for inclusion on the FY25 bill has already begun. Use our simple Advocacy Tool now to send a message to urge your elected officials to list Rett syndrome as a funding topic for FY25.

Advocate for Affordable Access
Your state elected officials often play a role in facilitating access to therapies, healthcare, and treatments, like the first-ever FDA-approved treatment for Rett syndrome, DAYBUE® (trofinetide). Get involved in raising Rett syndrome awareness by writing, emailing, or calling your State Congress members, and help move the needle toward overcoming Rett!

Give to Support Our Mission
Our work of advocating for families and strategically funding innovative research is only possible thanks to the many donors who make our mission possible. Give now to help us continue our efforts on behalf of every Rett family.

Thank you to The Conafay Group who donate their time and resources to advance our advocacy efforts. Thank you for your continued support of IRSF and the families we serve.

If you have any questions about how we advocate for families or want to talk about ways you can get involved, don’t hesitate to contact me at [email protected].