CURE SYNGAP1 Legislative Advocacy Efforts On Behalf Of Those Living With SYNGAP1

Overview (from EveryLife Foundation)

Do you have a Medicaid story to share? 

As part of the EveryLife Foundation for Rare Diseases’ initiative to protect the rare disease community’s access to Medicaid, we are asking advocates to share their Medicaid stories in their home state. We are aiming to collect at least two stories per state and territory to use for our efforts to retain Medicaid funding. Your story will be archived for future use in social media, communications with state legislatures, and even in pieces shared with news outlets. Of course, should we want to use your story, we will reach out before anything is shared publicly, and you will have final approval of what is included. Please be sure to include your contact information so we can streamline the approval process.

Join the Rare Disease Community Petition led by the EveryLife Foundation to urge Congress to sustain strong federal leadership, biomedical research funding, and public health support.

For decades, Congress has championed policies ensuring that no disease is too rare to deserve timely diagnosis, healthcare access, and treatment opportunities. However, recent executive actions are threatening critical programs that impact patients, families, researchers, and clinicians—jeopardizing the progress we’ve fought so hard to achieve.

The removal of key experts at NIH, FDA, and CDC, combined with federal funding restrictions, risks halting scientific advancements and delaying life-saving treatments. Even a short disruption could have devastating consequences for the 30 million Americans living with rare diseases.

Take Action Now!

• Sign the petition to demand steady and robust federal support
• Protect the future of biomedical research and healthcare access
• Encourage Congress and the Administration to prioritize rare disease policy

Help us ensure continued progress. Add your name today

Medicaid is a vital program for many individuals with SYNGAP1-Related Disorders (SRD) and other intellectual and developmental disabilities. Children and adults with SRD depend on Medicaid for essential healthcare, treatments, and other critical services that improve their quality of life. If Medicaid is reduced, vital care will vanish. If we don’t take action now, children and adults with disabilities could lose access to these services—some as early as April. 

Click below to access the Epilepsy Foundation website to contact your legislators to share why Medicaid is important for your loved one and everyone with epilepsy. This is an urgent request as we expect Congress to vote on the updated budget very soon!

Overview (from EveryLife Foundation)

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. 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.

See the full statement here.

A program of EveryLife Foundation for Rare Diseases, RDLA issues a monthly newsletter filled with Capitol Hill Updates, State Policy Highlights, and more. Read the latest, then sign up to receive updates straight to your inbox. Be informed so you can take action!

This multi-day event, hosted by the Rare Disease Legislative Advocates (a program of the EveryLife Foundation for Rare Diseases), brings together rare disease advocates from across the country to make their voices heard by their Members of Congress. Participants are educated on policy proposals impacting the rare disease community and provided opportunities to advocate for policy changes directly to their Members of Congress.

CURE SYNGAP1 Board members, staff, and community members will be attending this year’s event on February 24-26 in Washington, D.C.

Seventeen states (Alaska, Alabama, Arkansas, Florida, Georgia, Indiana, Iowa, Kansas, Louisiana, Missouri, Montana, Nebraska, South Carolina, South Dakota, Texas, Utah, and West Virginia) filed a lawsuit against the federal Health and Human Services Department about 504 protections (court filing linked here). It is publicized as a request to remove gender dysphoria as a protected disability under section 504 of the Rehabilitation Act of 1973. However, on the final page of the filing, the plaintiffs ask the court to order that section 504 be declared unconstitutional, not gender dysphoria alone, and that the states be protected from having to enact section 504 anti-discrimination policy.
 
How this would be detrimental to the disability community:

1. If the court makes orders that section 504 is unconstitutional, it could pave the way for those state legislatures to remove the statute and its protections for students and employees with disabilities.
2. A court order that section 504 is unconstitutional in this federal case can be used as precedent by other states seeking to remove anti-discrimination protections for people with disabilities.

Why this matters right now: 
 
The deadline for case briefs is February 25, 2025. This means there is still time to ask our elected representatives to file briefs in support of section 504. 
 
What we can do: 

1. Residents outside the 17 states in the lawsuit: Call and/or write to your state Attorney General and local elected officials, to request they take an official stand in this lawsuit to protect 504 provisions in this country. 
2. Residents of the 17 states in the lawsuit: Call and/or write to your state Attorney General and local elected officials. You can use the TAKE ACTION button below to link to a tool to help you find your Attorney General’s contact information. Tell them you oppose their request for section 504 to be declared unconstitutional. Consider sharing a personal story of how that would impact you or your loved one(s). Request that they change their position.
3. Ask family and friends to contact their state officials and Attorneys General, too.

Documents: These can be shared with elected officials and/or friends and family in your requests for action.

• HHS Section 504 Fact Sheet – a fact sheet about section 504 and what it does. Click this link to view or download.
• HHS Rehabilitation Act Complaint – the states’ court filing. Page 42 (d) and (e) are the requests for court orders about section 504 as a whole. Elected officials can refer to the case specifics when they file a brief. Click this link to view or download.

*Thank you to We Are Brave Together for compiling this information.

The Congressional Rare Disease Caucus is a forum for Members of Congress to voice constituent concerns, collaborate on ideas, facilitate conversations between the medical and patient community and build support for legislation that will improve the lives of people with rare diseases.

A full list of members of the caucus can be found here. If your Representative and/or Senator is not shown, please tell them why it’s important to you for them to join by clicking the button below.

A caucus is a group of bi-partisan legislators that join together to support a specific cause. This newly formed Epilepsy Caucus will help to promote legislation that will benefit the epilepsy community, so we need to get as many members as possible to join.

Click below to check to see if your legislators are already members of the caucus. If not, you can use the form to send a message to ask them to join!

There is a new effort underway to create a National Plan for the Epilepsies. This legislation, if passed, would direct the federal government to develop a National Plan for Epilepsy to prevent, diagnose, treat, and cure the epilepsies and improve the well-being of people with epilepsy and their families. We support anything that would bring more attention, research, and resources to the epilepsy community. 

The AKAC Act streamlines out-of-state Medicaid provider enrollment, reducing delays and
administrative burdens for doctors. Many rare disease patients on Medicaid must travel for
specialist care not available in their home state. However, to obtain care in another state, the
doctors must be enrolled in the patient’s home state’s Medicaid program, creating long delays in
provider enrollment that often slow or block access.

This bill would make it easier for patients to see providers out of state because it would set up a framework for doctors to more easily accept Medicaid plans from other states. For our community, that means potentially not having to pay out of pocket to see a SYNGAP1 specialist in a different state.

Despite bipartisan support and the inclusion of the bill in the House’s version of H.R.1, the bill
was dropped from the 2024 year-end package. It has since been reintroduced in both chambers. We will share an action alert once available.

The Newborn Screening Saves Lives Act (NBSSLA) established national guidelines in 2008, leading to required screening for at least 32 treatable conditions in all states and D.C. Though not reauthorized since 2019, Congress continued funding CDC, NIH, and HRSA programs — but that could change.

Last week, the committee that reviews evidence and provides advice on the Recommended Uniform Screening Panel (RUSP), the Advisory Committee for Heritable Disorders for Newborns and Children (ACHDNC), was terminated, effective immediately.

On November 13, 2025, The Philadelphia Inquirer reported on a paper urging Pennsylvania legislators to support the Senate mark for appropriations to the NIH for fiscal year 2026. In the letter, CURE SYNGAP1 joined several major Philadelphia-area research institutions (Pennsylvania Ad Hoc NIH Funding Advocacy Coalition) to urge key Pennsylvania representatives in Congress to reject proposed cuts to federal research dollars.

A similar letter was sent on August 20, 2025, also as reported by The Philadelphia Inquirer.

This bill would establish the California Epilepsy Program within the California Department of Public Health to conduct epidemiological assessments of epilepsy and seizures, focusing on incidence and prevalence. This legislation would significantly improve the state’s ability to understand and address the burden of epilepsy and seizures state-wide and work toward solutions to improve outcomes for Californians with epilepsy.