Expressing support for the designation of April 5, 2024, as "Barth Syndrome Awareness Day".

This resolution supports the designation of Barth Syndrome Awareness Day. Barth Syndrome is a rare, life-threatening genetic disorder of lipid metabolism that primarily affects males.

[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[H. Res. 1025 Introduced in House (IH)]

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118th CONGRESS
  2d Session
H. RES. 1025

  Expressing support for the designation of April 5, 2024, as ``Barth 
                       Syndrome Awareness Day''.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                           February 20, 2024

 Mr. Tonko (for himself, Mr. Bilirakis, and Mr. Norman) submitted the 
following resolution; which was referred to the Committee on Energy and 
                                Commerce

_______________________________________________________________________

                               RESOLUTION


 
  Expressing support for the designation of April 5, 2024, 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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