Members of the Connecticut Team outside the Capitol Building. From left to right: Rachel, Jonathan, Kelly and me.

In 1995, at age 10, I was diagnosed with congenital myasthenic syndromes. This diagnosis eventually led me down a path of advocacy and raising awareness of rare diseases. You can read my story in this blog post.

In 2019 I attended my first Rare Disease Week on Capitol Hill with the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA). I was able to attend again this year and would love to share more about what it’s like to be a rare disease advocate.

Rare Disease Week takes place in Washington, D.C., where patients, advocates, healthcare professionals, researchers, and policymakers come together to raise awareness about rare diseases and advocate for policies that support research, treatment, and access to care for individuals with rare diseases.

During Rare Disease Week on Capitol Hill, participants engage in activities such as advocacy meetings with members of Congress, educational events, networking opportunities, and awareness campaigns. The goal of the event is to highlight the unique challenges faced by individuals with rare diseases and to urge policymakers to take action to address these challenges.

In 2023 and 2024, I was a team coordinator for Connecticut, alongside Kelly Considine, another rare patient advocate.

The week kicked off on Sunday evening, February 25, 2024, with a cocktail reception and documentary screening, as well as time to meet others in RDLA.

Monday was a full day of conferences, where different organizations and individuals shared research, stories, and recent breakthroughs for the rare community. We also met our Connecticut team, and Kelly and I spent some time with our group going over our legislative asks and making sure everyone was comfortable with speaking and sharing their unique story.

Tuesday was our Hill Day and lobby day. We were on the Hill from 11:00 a.m. to about 3:30 p.m. The Connecticut team met with Senator Richard Blumenthal, Representative Joe Courtney, Representative Jhana Hayes, Representative Chris Murphy, and Congresswoman Rosa DeLauro.

Drea, another Rare Advocate volunteer who lives with MG, joined several events on the Hill to represent those with myasthenia and myasthenic syndromes.

When meeting in these respective offices, we usually end up meeting with the Legislative Director (LD) or Legislative Assistant (LA). The LD/LA listen to each personal rare story from the team of advocates, take notes, and ask questions. We also request legislative asks.

What’s a legislative ask? These are the specific topics we bring to our reps. We may ask them to sponsor bills or join the Rare Disease Congressional Caucus if they are not already involved. This year, the Connecticut team focused on the Safe Step Act and the Protect Rare Act. We also asked them to please join the Rare Disease Congressional Caucus if they are not already.

The Safe Step Act requires a group health plan to establish an exception to medication step-therapy protocol in specified cases. A medication step-therapy protocol establishes a specific sequence in which prescription drugs are covered by a group health plan or a health insurance issuer.
The PROTECT Rare Act will allow Medicare and Medicaid to cover the cost of medication or treatment that doctors think would benefit their patients if appropriate, providing flexibility to Americans living with rare diseases.

The Rare Disease Caucus is a group of lawmakers within a legislative body who have a specific interest in advocating for policies and legislation related to rare diseases. These caucuses typically work to raise awareness about rare diseases, support research funding, improve access to treatment and care, and promote policies that benefit individuals and families affected by rare diseases. By bringing together lawmakers with a shared interest in rare diseases, these caucuses can help advance initiatives that may not receive as much attention in broader legislative discussions.

Caucuses like these are important to give a voice to those affected by rare diseases and to drive policy changes to address the unique challenges faced by individuals living with these conditions. The work of rare disease caucuses can lead to increased research, improved healthcare services, and better support for patients and families dealing with rare diseases. Our goal is to get as many Legislators as possible join the caucus.

Rare Disease Week is a phenomenal event for the rare disease community and something I would encourage others with a rare disease to look into being involved with, whether it be in person or virtually. Joining RDLA is your first step. It’s a fantastic group.

So few people know what congenital myasthenic syndrome and myasthenia gravis are, but with advocating and raising awareness, we can bring more knowledge to world.

A disease may be rare, but hope should not be. While we wait for a cure, hope will prevail.

Get involved! If you’d like to advocate on behalf of people with myasthenia gravis and other myasthenic syndromes, get in touch with the MGFA. Email us at mgfa@myasthenia.org. Check out the website for the Rare Disease Legislative Advocates (RDLA) for more information on joining.