Bill Summary
The resolution expresses support for designating April 5, 2025, as "Barth Syndrome Awareness Day." Barth syndrome is a rare genetic disorder primarily affecting males, characterized by severe health issues such as heart and skeletal muscle abnormalities, low white blood cell counts, and growth delays. The disorder has a low incidence rate, with fewer than 10 new cases identified in the U.S. each year, and it can be life-threatening, especially in early childhood.
The resolution highlights the challenges faced by individuals with Barth syndrome, including difficulties in diagnosis, limited treatment options, and the need for specialized medical care. It acknowledges the critical need for research and development of treatments, as no FDA-approved therapies currently exist. The resolution also notes the role of the Barth Syndrome Foundation in raising awareness and advocating for research.
By supporting "Barth Syndrome Awareness Day," the House of Representatives aims to improve public understanding of the disorder, promote early and accurate diagnosis, and encourage advancements in research and treatment options for those affected by Barth syndrome.
Possible Impacts
The resolution designating April 5, 2025, as "Barth Syndrome Awareness Day" could affect people in the following ways:
1. **Increased Awareness and Understanding**: The designation of a specific day for Barth syndrome awareness can lead to greater public awareness of this rare genetic disorder. This increased awareness may help individuals recognize symptoms more readily, potentially leading to earlier diagnosis and intervention for affected individuals. Families may feel less isolated as the condition gains visibility, fostering a sense of community and support.
2. **Advocacy for Research and Funding**: By recognizing Barth Syndrome Awareness Day, the resolution may encourage advocacy efforts aimed at increasing funding for research and development of treatments for Barth syndrome. This could lead to advancements in medical research, potentially resulting in new therapies that improve the quality of life for individuals with the condition and perhaps even finding a cure.
3. **Improved Access to Care and Resources**: The resolution emphasizes the need for improved access to specialized healthcare services for individuals with Barth syndrome. By raising awareness, it may encourage healthcare providers and institutions to allocate resources towards the training of specialists and the establishment of more treatment centers. This could lead to better healthcare outcomes, as patients would have access to interdisciplinary care tailored to their unique needs, thereby reducing the challenges they currently face in obtaining appropriate treatment.
[Congressional Bills 119th Congress]
[From the U.S. Government Publishing Office]
[H. Res. 209 Introduced in House (IH)]
<DOC>
119th CONGRESS
1st Session
H. RES. 209
Expressing support for the designation of April 5, 2025, as ``Barth
Syndrome Awareness Day''.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
March 10, 2025
Mr. Tonko (for himself, Mr. Bilirakis, Ms. Matsui, Mr. Norman, Mrs.
Trahan, Mr. Wilson of South Carolina, and Mr. Auchincloss) submitted
the following resolution; which was referred to the Committee on Energy
and Commerce
_______________________________________________________________________
RESOLUTION
Expressing support for the designation of April 5, 2025, as ``Barth
Syndrome Awareness Day''.
Whereas Barth syndrome is a rare, life-threatening, genetic disorder which
primarily affects males;
Whereas Barth syndrome is caused by a mutation in the tafazzin gene (TAFAZZIN,
also called G4.5), resulting in an inborn error of phospholipid
metabolism, affecting many systems of the body;
Whereas Barth syndrome is a multisystem disorder with characteristics including
abnormalities of heart and skeletal muscle, low levels of certain white
blood cells called neutrophils that help to fight bacterial infections,
and delays in growth;
Whereas with probably fewer than 10 new Barth infants identified each year in
the United States, the incidence of Barth syndrome is likely only 1 in
every 300,000 to 400,000 births and could be as few as 1.5 in 1 million;
Whereas globally there are approximately 300 individuals diagnosed with Barth
syndrome, and, in the United States, there are fewer than 150
individuals diagnosed with Barth syndrome;
Whereas Barth syndrome can be fatal in childhood due to heart failure or
uncontrollable infection, with approximately 50 percent of deaths due to
Barth syndrome occurring within the first year of life and 85 percent
before the fifth year of life;
Whereas individuals with Barth syndrome who survive to adulthood often have a
severely limited life expectancy;
Whereas individuals with Barth syndrome, like many other rare disorders,
experience challenges with obtaining a diagnosis, limited treatment
options, and difficulty finding and accessing treatment centers and
physicians with expertise in Barth syndrome;
Whereas, because the disorder affects multiple systems of the body, a patient
with Barth syndrome often requires access to care from experts across a
range of medical specialties;
Whereas the Barth Syndrome Clinic at Kennedy Krieger Institute is the only
interdisciplinary clinic dedicated to the diagnosis and treatment of
Barth syndrome in the United States;
Whereas, to date, there is no treatment for Barth syndrome that is approved by
the Food and Drug Administration (FDA);
Whereas there is a critical need for research and development to advance
treatments for Barth syndrome;
Whereas, as a result of the Orphan Drug Act, there have been important advances
in research on and treatment for rare diseases, including development
efforts in Barth syndrome;
Whereas the FDA established the Accelerated Approval Pathway in 1992 and
Congress codified the pathway in 2012;
Whereas the Accelerated Approval Pathway is an important pathway for rare and
ultrarare diseases as it allows for ``earlier approval of drugs that
treat serious conditions, and fill an unmet medical need . . .'';
Whereas Congress and the FDA have affirmed the importance of incorporating the
patient perspective throughout the drug review process through the FDA's
Patient-Focused Drug Development program;
Whereas the Barth Syndrome Foundation, a nonprofit organization established in
2000 to accelerate progress through collaboration between families and
scientists, is dedicated to educating, advancing treatments, and finding
a cure for Barth syndrome;
Whereas the Barth Syndrome Foundation sponsors ``Barth Syndrome Awareness Day''
in the United States to increase public awareness and generate
additional support for Barth syndrome; and
Whereas ``Barth Syndrome Awareness Day'' is expected to be observed in the
United States for years to come, providing hope and information for
patients, caregivers, and families around the country: Now, therefore,
be it
Resolved, That the House of Representatives--
(1) supports the designation of ``Barth Syndrome Awareness
Day''; and
(2) recognizes the importance of, with respect to Barth
syndrome--
(A) improving awareness;
(B) encouraging accurate and early diagnosis;
(C) advancing research;
(D) developing new treatments, diagnostics, and
cures; and
(E) identifying regulatory pathways for drug
development of ultrarare diseases like Barth syndrome.
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