Current Actions

Urge Your U.S. Senator to Oppose Steep Budget Cuts to the National Institutes of Health (NIH)!

Wed, 30 Jul 2025 04:00:00 GMT

The Senate Appropriations Committee is considering the Administration’s request to cut NIH funding by nearly 40 percent for its fiscal year 2026 budget which could harm critical, lifesaving work at the National Institutes of Health (NIH). Please ask your members of Congress to support the rare disease community by asking for robust funding for NIH!

Take action today!

Tell Policymakers to Support our Federal Newborn Screening System

Wed, 23 Apr 2025 04:00:00 GMT

September is Newborn Screening Awareness Month, and your policymakers need to hear from you! Earlier this year, the Administration eliminated the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), and many federal newborn screening activities may be impacted by other changes at the Department of Health and Human Services (HHS). The ACHDNC and these federal programs are a critical element of the U.S. newborn screening system, supporting state newborn screening programs and playing a central role in the process of adding conditions to the federal Recommended Uniform Screening Panel (RUSP), federal guidelines outlining what serious rare conditions states should screen for. Reach out to your policymakers to share why newborn screening matters to you and urge them to protect our federal newborn screening system.

Urge Your Lawmakers to Get Rare Pediatric Disease Priority Review Voucher (PRV) Program Reauthorization Across the Finish Line!

Mon, 07 Apr 2025 04:00:00 GMT

Members of Congress have the opportunity to advance important legislation that supports rare disease innovation for children. Since 2012, the FDA has awarded companies that develop life-saving and novel therapies for children with rare diseases with priority review vouchers (PRVs) to help spur innovation in this critical area. The Give Kids a Chance Act (H.R. 1262/ S. 932) would reauthorize the Rare Pediatric Disease PRV program through 2029. However, legislation extending this program must pass swiftly to ensure this important tool is preserved for addressing the significant unmet treatment needs that exist in the pediatric rare disease population.

We need your help funding the Rare Disease Advisory Council in California!

Thu, 06 Mar 2025 05:00:00 GMT

AB 2613 (Zbur) was passed unanimously by both the Assembly and Senate, and signed into law by California Governor Newsom in September 2024, creating the Jacqueline Marie Zbur Rare Disease Advisory Council (RDAC). This Council will give California rare disease patients, families, caregivers, providers, and other stakeholders an opportunity to make formal recommendations to state decision-makers on how to improve public policy for our community. However, the RDAC must receive funding before it can become a reality! Californians, contact your Assemblymember on the Assembly Budget Committee today to share why funding the Jacqueline Marie Zbur Rare Disease Advisory Council is important and ask for their support!

Join the Rare Action Network

Thu, 11 Sep 2025 04:00:00 GMT

Members of the Rare Action Network are part of 30+ million person community working towards improving the lives of patients with rare diseases. RAN members will positively improve the quality of life for rare disease patients and their families by ensuring rare diseases become a national priority through policy, education and awareness. JOIN TODAY!