IV 

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; 


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•HRES 1025 IH 

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); 


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•HRES 1025 IH 

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 

1 Resolved, That the House of Representatives— 


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•HRES 1025 IH 

1 (1) supports the designation of ‘‘Barth Syn2 drome Awareness Day’’; and 

3 (2) recognizes the importance of, with respect 

4 to Barth syndrome— 

5 (A) improving awareness; 

6 (B) encouraging accurate and early diag7 nosis; 

8 (C) advancing research; 

9 (D) developing new treatments, 

10 diagnostics, and cures; and 

11 (E) identifying regulatory pathways for 

12 drug development of ultrarare diseases like 

13 Barth syndrome. 

Æ 


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