110TH CONGRESS 
2D SESSION 
H. R. 5265 
AN ACT 
To amend the Public Health Service Act to provide for 
research with respect to various forms of muscular dys-
trophy, including Becker, congenital, distal, Duchenne, 
Emery-Dreifuss 
facioscapulohumeral, 
limb-girdle, 
myotonic, and oculopharyngeal, muscular dystrophies. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled,
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Paul D. Wellstone 
Muscular Dystrophy Community Assistance, Research, 
and Education Amendments of 2008’’. 
SEC. 2. FINDINGS. 
The Congress finds as follows: 
(1) The muscular dystrophies are devastating 
diseases that have a significant impact on quality of 
life—not only for the individual who experiences its 
painful symptoms and resulting disability, but also 
for family members and caregivers. 
(2) DMD is the most common lethal genetic 
disorder of childhood worldwide, affecting approxi-
mately 1 in every 3,500 boys born each year around 
the globe. It is characterized by a rapidly progressive 
muscle weakness that almost always results in death 
from respiratory or cardiac failure, typically in the 
late teens or twenties. 
(3) Myotonic muscular dystrophy is the second 
most prominent form of muscular dystrophy and the 
type most commonly found in adults affecting an es-
timated 1 in 8,000 people. However, it can affect 
people of any age—from birth to old age. Described 
as the most variable disease known in medicine, it 
is multi-systemic and can cause not only muscle at-
rophy and myotonia, but also serious cardiac, res-
piratory, endocrine, gastrointestinal, skeletal and 
central nervous system complications, as well as 
problems with the eyes, teeth and hair. As it passes 
from one generation to the next, it generally worsens 
with earlier onset. Congenital myotonic muscular 
dystrophy is the most severe form of myotonic mus-
cular dystrophy affecting infants and causing severe 
cognitive delays. It often causes sudden death; how-
ever, others can live for many years with this slowly 
degenerative disorder. 
(4) Facioscapulohumeral muscular dystrophy 
(referred to in this section as ‘‘FSHD’’) is the sec-
ond most prevalent adult muscular dystrophy and 
the third most prevalent muscular dystrophy of men, 
women and children. It is inherited genetically and 
has an estimated incidence of 1 in 20,000 persons. 
Many leading FSHD scientists note that the preva-
lence may be three times higher due to undiagnosed 
and misdiagnosed cases. FSHD, affecting between 
15,000 to 40,000 persons, causes a lifelong progres-
sive and severe loss of all skeletal muscles gradually 
bringing weakness and reduced mobility. It is geneti-
cally transmitted to children, can occur spontane-
ously, and may affect entire families. Persons with 
FSHD may also experience hearing loss, vision prob-
lems and respiratory insufficiency; some may become 
severely physically disabled and spend decades in a 
wheelchair and on a ventilator. FSHD is caused by 
a novel epigenetic phenomenon not found in other 
forms of muscular dystrophy and is caused by a con-
traction of repetitive DNA previously thought to be 
‘‘junk DNA’’. The unique epigenetic structure of 
FSHD is unprecedented in other muscular dys-
trophies and genetic disorders and demands novel 
approaches and new research groups. Understanding 
this mechanism will have great benefit to other areas 
of biomedical research including cancer and other 
disease of epigenetic origin. 
(5) Congenital muscular dystrophies represent a 
group of distinct diseases, which begin at birth, with 
varying severity and involvement of both muscle 
strength and brain. These diseases often lead to pre-
mature infant death, or severely disabled young chil-
dren who require 24-hour care given their develop-
mental delay compounded by muscle weakness. 
Other children live to young adulthood and typically 
require the use of a wheelchair for mobility. 
(6) Forms of muscular dystrophy affecting chil-
dren and adults include Becker, congenital, distal, 
Duchenne, 
Emery-Dreifuss, 
facioscapulohumeral,
limb-girdle, myotonic, and oculopharyngeal muscular 
dystrophies. The limb-girdle muscular dystrophies 
are of 15 known different types. 
(7) Each of the muscular dystrophies, though 
distinct in progressivity and severity of symptoms, 
has a devastating impact on hundreds of thousands 
of children and adults throughout the United States 
and worldwide, as well as imposes severe physical 
and economic burdens on those affected. In many of 
the muscular dystrophies, there are associated med-
ical problems arising from pulmonary issues, res-
piratory insufficiency, cardiomyopathy, which in 
many cases is the cause of death for persons with 
muscular dystrophy. 
(8) In the 5 years since enactment of the Mus-
cular Dystrophy Community Assistance, Research 
and Education Amendments of 2001 (MD–CARE 
Act) and due directly to the momentum established 
by the MD–CARE Act, progress has been made in 
the battle against the Muscular Dystrophies. 
(9) Investments made by the Federal Govern-
ment as a result of the MD–CARE Act include the 
creation 
of 
the 
MD 
Coordinating 
Committee 
(MDCC), the development of the MDCC Action 
Plan, establishment of 6 Paul D. Wellstone Mus-
cular Dystrophy Cooperative Research Centers (co- 
funded, in part, by a national non-profit health orga-
nization), development of the Muscular Dystrophy 
Surveillance, Tracking and Research Network (MD 
STARnet), and the launch of a comprehensive edu-
cation and outreach initiative. 
(10) In the past few years, the NIH program 
in translational research in muscular dystrophy has 
grown significantly and funded a number of large- 
scale projects to further the development of thera-
pies for muscular dystrophy. As part of this pro-
gram, the National Institute of Neurological Dis-
orders and Stroke (NINDS) and the National Insti-
tute of Arthritis and Musculoskeletal and Skin Dis-
eases (NIAMS) of the National Institutes of Health 
(NIH) awarded a $15.4 million, five-year cooperative 
agreement to develop new small molecule drugs for 
the treatment of Duchenne muscular dystrophy 
(DMD) and potentially other forms of muscular dys-
trophy as well. The project is a unique research col-
laboration between private, public, and non-profit 
partners to build upon previous research and dis-
covery work originally initiated by non-profit part-
ners to identify new treatments for muscular dys-
trophy. Also through the translational program,