IV
119THCONGRESS
1
STSESSION H. RES. 209
Expressing support for the designation of April 5, 2025, as ‘‘Barth Syndrome
Awareness Day’’.
IN THE HOUSE OF REPRESENTATIVES
MARCH10, 2025
Mr. T
ONKO(for himself, Mr. BILIRAKIS, Ms. MATSUI, Mr. NORMAN, Mrs.
T
RAHAN, Mr. WILSONof South Carolina, and Mr. AUCHINCLOSS) sub-
mitted 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 skel-
etal 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 209 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 di-
agnosed 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 approxi-
mately 50 percent of deaths due to Barth syndrome oc-
curring 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 di-
agnosis, limited treatment options, and difficulty finding
and accessing treatment centers and physicians with ex-
pertise in Barth syndrome;
Whereas because the disorder affects multiple systems of the
body, a patient with Barth syndrome often requires ac-
cess to care from experts across a range of medical spe-
cialties;
Whereas the Barth Syndrome Clinic at Kennedy Krieger In-
stitute 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 209 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 Path-
way 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 impor-
tance of incorporating the patient perspective throughout
the drug review process through the FDA’s Patient-Fo-
cused Drug Development program;
Whereas the Barth Syndrome Foundation, a nonprofit orga-
nization established in 2000 to accelerate progress
through collaboration between families and scientists, is
dedicated to educating, advancing treatments, and find-
ing a cure for Barth syndrome;
Whereas the Barth Syndrome Foundation sponsors ‘‘Barth
Syndrome Awareness Day’’ in the United States to in-
crease 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, pro-
viding hope and information for patients, caregivers, and
families around the country: Now, therefore, be it
Resolved, That the House of Representatives— 1
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•HRES 209 IH
(1) supports the designation of ‘‘Barth Syn-1
drome Awareness Day’’; and 2
(2) recognizes the importance of, with respect 3
to Barth syndrome— 4
(A) improving awareness; 5
(B) encouraging accurate and early diag-6
nosis; 7
(C) advancing research; 8
(D) developing new treatments, 9
diagnostics, and cures; and 10
(E) identifying regulatory pathways for 11
drug development of ultrarare diseases like 12
Barth syndrome. 13
Æ
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