[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[H. Res. 1307 Introduced in House (IH)]
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118th CONGRESS
2d Session
H. RES. 1307
Expressing support for the designation of June 20, 2024, as ``World
FSHD Day''.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
June 18, 2024
Mrs. Dingell (for herself and Mr. Kelly of Pennsylvania) submitted the
following resolution; which was referred to the Committee on Energy and
Commerce
_______________________________________________________________________
RESOLUTION
Expressing support for the designation of June 20, 2024, as ``World
FSHD Day''.
Whereas facioscapulohumeral muscular dystrophy (FSHD) is a genetic neuromuscular
disease (NMD) that leads to the weakening of skeletal muscles;
Whereas FSHD's name comes from the typical pattern of muscle weakness at onset,
beginning with the face (facio), shoulder girdle (scapulo), and upper
arms (humeral);
Whereas FSHD Type 1 is the more common form of FSHD, accounting for
approximately 95 percent of FSHD cases; results from deletions in the
D4Z4 region on chromosome 4; and leads to abnormal expression of the
DUX4 gene;
Whereas, approximately 5 percent of FSHD cases, known as FSHD Type 2, are linked
to mutations on a gene called SMCHD1, located on chromosome 18;
Whereas there are still many FSHD cases of unknown genetic cause;
Whereas FSHD is an inherited condition that can affect many family members
across generations;
Whereas FSHD is genetically transmissible in an autosomal dominant fashion,
meaning that an affected parent has a 50 percent chance of passing the
genetic defect on to each child;
Whereas 30 percent of new FSHD patients have no prior family history of the
disease and are affected as a result of congenital spontaneous genetic
mutation;
Whereas FSHD exists worldwide, affects both sexes equally, and has no particular
racial, geographic, or ethnic distribution;
Whereas an estimated 1 in 8,000 individuals, or 41,487 Americans, are living
with FSHD;
Whereas FSHD symptoms can develop at any age and can differ in the initial
pattern of muscle weakness;
Whereas weakness in abdominal muscles is common and can lead to lordosis, an
exaggerated curve in the lower spine;
Whereas the loss of upper body mobility is a debilitating symptom of FSHD that
significantly impacts patients' ability to perform daily tasks necessary
to care for themselves;
Whereas lower leg weakness often occurs and can lead to a condition called foot
drop, where the foot stays down after pushing off when walking;
Whereas FSHD can reduce patients' ability to work and earn a living;
Whereas asymmetrical muscle loss is a hallmark symptom of FSHD with an unknown
cause, and most patients observe that one arm, shoulder blade, or lower
leg is weakened while the other remains stronger;
Whereas about 20 percent of FSHD patients will become dependent on a wheelchair
or scooter;
Whereas patients with FSHD may develop progressive weakness of the respiratory
muscles or scoliosis, which can cause fatal respiratory insufficiency;
Whereas more than 70 percent of FSHD patients experience debilitating pain and
fatigue, which can severely limit daily activities;
Whereas the loss of facial expression and mobility, as well as others' lack of
understanding of FSHD, is emotionally distressing and can cause FSHD
patients to withdraw socially;
Whereas individuals with FSHD, like those with other rare disorders, experience
challenges in obtaining a diagnosis, with the average time to receive an
accurate FSHD diagnosis being 9 years;
Whereas genetic testing is needed to definitively diagnose FSHD, and further
investments in genetic testing are required to ensure access to testing
for all Americans;
Whereas, to date, the Food and Drug Administration (FDA) has yet to approve any
treatments for FSHD;
Whereas, as a result of the Orphan Drug Act, there have been important advances
in research on and treatment for rare diseases, including efforts to
develop treatments for FSHD;
Whereas Congress and the FDA have affirmed the importance of incorporating
patient perspectives throughout the drug review process through
implementation of the 21st Century Cures Act, the HEART Act, and FDA
guidance;
Whereas the FDA's Patient-Focused Drug Development program is a critical
resource that allows patients and caregivers to educate the FDA and
other stakeholders on their lived experiences;
Whereas there is a critical need for research and development to advance
treatments for FSHD;
Whereas the FSHD Society, a nonprofit organization established in 1991, is
dedicated to increasing, engaging, and empowering stakeholders and
aggressively leveraging and expanding resources, with the goal of
developing treatments for FSHD by 2025 and eventually a cure;
Whereas, on June 29, 2020, the FSHD community held its first-ever externally led
patient-focused drug development meeting; and
Whereas FSHD patient advocacy organizations, research funding nonprofits,
biopharmaceutical industry partners, and other key stakeholders sponsor
World FSHD Day in the United States to increase public awareness and
generate additional support for FSHD: Now, therefore, be it
Resolved, That the House of Representatives--
(1) supports the designation of ``World FSHD Day''; and
(2) recognizes the importance of--
(A) improving awareness of and education about
facioscapulohumeral muscular dystrophy (in this
resolution referred to as ``FSHD'');
(B) encouraging accurate and early diagnosis of
FSHD through genetic screening;
(C) supporting and funding future biomedical and
scientific research to improve physical functioning and
quality of life for individuals living with FSHD;
(D) developing new treatments, diagnostics, and
cures for FSHD; and
(E) advancing programs and policies that support
individuals living with FSHD and their caregivers.
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