Expressing support for the designation of April 5, 2025, as "Barth Syndrome Awareness Day".
- Bill Number
- H.Res. 209
- Origin Chamber
- House
- Congress
- 119th Congress, Session 1
- Policy Area
- Health
- Status
- Introduced
- Latest Action
- 2025-03-10: Referred to the House Committee on Energy and Commerce.
- Last Updated
- 2026-02-19T16:40:58Z
AI-Generated Summary
Purpose
This resolution (H. Res. 209) expresses the House of Representatives' support for designating April 5, 2025, as "Barth Syndrome Awareness Day." Its main goal is to raise public awareness about Barth syndrome, a rare and life-threatening genetic disorder that primarily affects males, and to highlight the need for better diagnosis, research, and treatments.
Key Provisions
- Description of Barth Syndrome: Outlines the disorder's causes (a mutation in the tafazzin gene leading to errors in phospholipid metabolism), symptoms (heart and muscle abnormalities, low neutrophil levels increasing infection risk, growth delays), rarity (about 1 in 300,000–400,000 births in the U.S., with fewer than 150 diagnosed cases nationwide and around 300 globally), and challenges (high childhood mortality, limited treatment options, and need for multidisciplinary care).
- Support for Awareness Day: Endorses the Barth Syndrome Foundation's annual event to educate the public, generate support, and provide hope to patients and families.
- Recognition of Importance: Emphasizes advancing:
- Awareness and early diagnosis.
- Research and development of new treatments, diagnostics, and cures.
- Regulatory pathways for drugs targeting ultra-rare diseases (e.g., referencing the FDA's Accelerated Approval Pathway and Patient-Focused Drug Development program).
- Acknowledgment of Existing Efforts: Notes contributions from the Orphan Drug Act, the Barth Syndrome Clinic at Kennedy Krieger Institute (the only U.S. center dedicated to the disorder), and the Barth Syndrome Foundation (founded in 2000 to promote collaboration between families and scientists).
Significant Changes to Existing Law
This is a non-binding resolution, so it introduces no changes to existing laws or regulations. It serves as a symbolic statement of support rather than enforceable legislation.
Potential Impacts
- On Citizens: Increases public knowledge of Barth syndrome, potentially leading to earlier diagnoses for affected individuals (mostly young males) and better support for families facing high mortality risks (e.g., 50% of deaths in the first year of life).
- On Government Agencies: Indirectly encourages the FDA and other health agencies to prioritize rare disease research and patient perspectives in drug approvals, though no new mandates are created.
- On International Relations: Minimal impact, as the resolution focuses on U.S.-based awareness but notes global prevalence, which could foster international collaboration on rare disease research.
- Broader Effects: May inspire increased funding or policy focus on ultra-rare diseases, benefiting the small affected population without significant costs to the government.
Main Stakeholders Affected
- Patients and Families: Primarily males with Barth syndrome (fewer than 150 in the U.S.) and their caregivers, who face diagnosis delays, limited treatments, and lifelong challenges.
- Medical and Research Community: Physicians, specialists (e.g., in cardiology, immunology), researchers, and institutions like the Barth Syndrome Clinic and FDA, who are urged to advance diagnostics and therapies.
- Advocacy Groups: The Barth Syndrome Foundation, which organizes awareness efforts and pushes for cures.
- General Public: Benefits from heightened awareness, potentially reducing stigma and improving access to specialized care.
Notable Legal, Constitutional, or Political Implications
- Legal: No binding effects; it reaffirms existing frameworks like the Orphan Drug Act (which incentivizes rare disease drug development) and FDA pathways without altering them.
- Constitutional: None, as resolutions like this fall under Congress's free speech and informational roles without infringing on rights.
- Political: Represents bipartisan support (introduced by members from both parties) for rare disease advocacy, signaling congressional priority on underserved health issues. It could politically encourage future funding for research or influence FDA policies on patient input, but remains symbolic to avoid controversy over resource allocation.
This summary was generated by AI and may contain inaccuracies. Refer to the official source document for the authoritative text.
Sponsor
Cosponsors (14)
Rep. Bilirakis, Gus M. [R-FL-12], Rep. Matsui, Doris O. [D-CA-7], Rep. Norman, Ralph [R-SC-5], Rep. Trahan, Lori [D-MA-3], Rep. Wilson, Joe [R-SC-2], Rep. Auchincloss, Jake [D-MA-4], Rep. McGarvey, Morgan [D-KY-3], Rep. Davids, Sharice [D-KS-3], Rep. Biggs, Sheri [R-SC-3], Rep. McGuire, John [R-VA-5], Rep. Flood, Mike [R-NE-1], Rep. Latimer, George [D-NY-16], Rep. Guest, Michael [R-MS-3], Rep. Neguse, Joe [D-CO-2]
Recent Actions
- 2025-03-10: Referred to the House Committee on Energy and Commerce.
- 2025-03-10: Submitted in House
- 2025-03-10: Submitted in House
Bill Versions
- Expressing support for the designation of April 5, 2025, as "Barth Syndrome Awareness Day". — issued 2025-03-10 — PDF (4 pages)