I 
110TH CONGRESS 
2D SESSION 
H. R. 5265 
To amend the Public Health Service Act to provide for research with respect 
to various forms of muscular dystrophy, including Becker, congenital, 
distal, Duchenne, Emery-Dreifuss facioscapulohumeral, limb-girdle, myo-
tonic, and oculopharyngeal, muscular dystrophies. 
IN THE HOUSE OF REPRESENTATIVES 
FEBRUARY 7, 2008 
Mr. ENGEL (for himself and Mr. BURGESS) introduced the following bill; 
which was referred to the Committee on Energy and Commerce 
A BILL 
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, myo-
tonic, 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’’. 
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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 
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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 
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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. 
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(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, expansion of the NIH research portfolios, es-
tablishment of 6 Paul D. Wellstone Muscular Dys-
trophy Cooperative Research Centers (co-funded, in 
part, by a national non-profit health organization), 
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development of the Muscular Dystrophy Surveil-
lance, 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, 
three other major cooperative agreements have been 
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awarded for highly targeted therapy development 
projects in the muscular dystrophies. 
(11) Due to the initiatives made possible 
through the MD–CARE Act, national non-profit or-
ganizations have joined in model strategic collabora-
tions with academic research institutions, public 
funders of MD research, and industry to expand in-
vestments in MD research activities and to create 
new platforms for translational research. These have 
led to the development of the first potential thera-
pies for DMD, myotonic, facioscapulohumeral, limb- 
girdle and other conditions that are proceeding 
through clinical trials. 
(12) Advancements in care have helped prolong 
life and quality of life for patients with muscular 
dystrophy. 
(13) Notwithstanding these promising develop-
ments, the majority of the directions envisioned by 
the Action Plan for the Muscular Dystrophies, devel-
oped pursuant to the MD–CARE Act, have not been 
realized. 
Where 
recent 
momentum 
has 
been 
achieved, its sustainability is fragile and directly de-
pendent upon continued Federal support for the 
early phase planning and programs created through 
the MD–CARE Act. 
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(14) There remains a shortage of qualified re-
searchers in the field of muscular dystrophy re-
search. Many family physicians and health care pro-
fessionals still lack the knowledge and resources to 
detect and properly diagnose muscular dystrophy as 
early as possible, thus delaying management of 
symptoms 
in 
cases 
that 
go 
undetected 
or 
misdiagnosed. 
(15) As new understandings of the genetic basis 
for disease and potential treatment has emerged, the 
public and health care communities are in urgent 
need of education and outreach to ensure competent, 
informed engagement in genetic testing and coun-
seling and appropriate patient characterization so 
that patients are able to participate in new avenues 
of research and clinical trials. 
(16) As basic research into the muscular dys-
trophies points the way to new therapeutic targets, 
there is an urgent need to support the clinical re-
search infrastructure necessary to bring these thera-
peutic leads to human trials; these infrastructure 
needs include validated endpoints, current natural 
history studies, biomarkers, clinical research net-
works, patient registries and databases. 
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(17) In order to improve lives and develop effec-
tive treatments for individuals with muscular dys-
trophy, there must be improved communications and 
partnerships between patients, patient advocacy, re-
searchers, and clinical care providers. To that end, 
renewed effort to work together by all parties is a 
critical element for successful outcomes in the years 
to come. 
(18) Continued focus and investment are re-
quired to build on the current momentum, respond 
to public need, and ensure that federally funded re-
search and other innovation is translated to thera-
peutic targets as quickly as possible. 
SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINA-
TION OF ACTIVITIES OF NIH WITH RESPECT 
TO RESEARCH ON MUSCULAR DYSTROPHY. 
(a) TECHNICAL CORRECTION.—Section 404E of the 
Public Health Service Act (42 U.S.C. 283g) is amended 
by striking subsection (f) (relating to reports to Congress) 
and redesignating subsection (g) as subsection (f). 
(b) AMENDMENTS.—Section 404E of the Public 
Health Service Act (42 U.S.C. 283g) is amended— 
(1) in subsection (a)(1), by inserting ‘‘the Na-
tional Heart, Lung, and Blood Institute,’’ after ‘‘the 
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Eunice Kennedy Shriver National Institute of Child 
Health and Human Development,’’; 
(2) in subsection (b)(1), by adding at the end 
of the following: ‘‘Such centers of excellence shall be 
known as the ‘Paul D. Wellstone Muscular Dys-
trophy Cooperative Research Centers’.’’; and 
(3) by adding at the end the following: 
‘‘(g) CLINICAL RESEARCH.—The Coordinating Com-
mittee shall give special consideration to the urgent need 
to enhance the clinical research infrastructure required to 
test emerging therapies for the various forms of muscular 
dystrophy by prioritizing the achievement of the goals re-
lated to this topic in the plan under subsection (e)(1). 
‘‘(h) AUTHORIZATION OF APPROPRIATIONS.—There 
are authorized to be appropriated to carry out this section 
such sums as may be necessary for each of fiscal years 
2008 through 2012.’’. 
SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF 
CDC WITH RESPECT TO EPIDEMIOLOGICAL 
RESEARCH ON MUSCULAR DYSTROPHY. 
Section 317Q of the Public Health Service Act (42 
U.S.C. 247b–18) is amended— 
(1) by redesignating subsection (d) as sub-
section (f); and 
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(2) by inserting after subsection (c) the fol-
lowing: 
‘‘(d) DATA.—In carrying out this section, the Sec-
retary shall ensure that any data on patients that is col-
lected as part of the Muscular Dystrophy STARnet (under 
a grant under this section) is regularly updated to reflect 
changes in patient condition over time, particularly with 
respect to any improvements realized through patient ad-
herence to care considerations or utilization of a treatment 
or therapy. 
‘‘(e) REPORTS AND STUDY.— 
‘‘(1) ANNUAL
REPORT.—Not later than 18 
months after the date of the enactment of the Paul 
D. Wellstone Muscular Dystrophy Community As-
sistance, Research, and Education Amendments of 
2008, and annually thereafter, the Director of the 
Centers for Disease Control and Prevention shall 
submit to the appropriate committees of the Con-
gress a report— 
‘‘(A) concerning the activities carried out 
by MD STARnet site funded under this section 
during the year for which the report is pre-
pared; 
‘‘(B) containing the data collected and 
findings derived from the MD STARnet sites 
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each fiscal year (as funded under a grant under 
this section during fiscal years 2008 through 
2012); and 
‘‘(C) that every 2 years outlines prospec-
tive data collection objectives and strategies. 
‘‘(2) TRACKING HEALTH OUTCOMES.—The Di-
rector of the Centers for Disease Control and Pre-
vention shall provide prospective health outcome 
data on the health and survival of people with mus-
cular dystrophy.’’. 
SEC. 5. INFORMATION AND EDUCATION. 
Section 5 of the Muscular Dystrophy Community As-
sistance, Research and Education Amendments of 2001 
(42 U.S.C. 247b–19) is amended— 
(1) by redesignating subsection (c) as sub-
section (d); and 
(2) by inserting after subsection (b) the fol-
lowing: 
‘‘(c) REQUIREMENTS OF CDC.—In carrying out this 
section, the Director of the Centers for Disease Control 
and Prevention shall— 
‘‘(1) partner with leaders in the muscular dys-
trophy patient community; and 
‘‘(2) widely disseminate the Duchenne-Becker 
muscular dystrophy care considerations as broadly 
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as possible, including through partnership opportuni-
ties with the muscular dystrophy patient commu-
nity.’’. 
SEC. 6. STANDARDS OF CARE. 
Part A of title IX of the Public Health Service Act 
(42 U.S.C. 299 et seq.) is amended by adding at the end 
the following: 
‘‘SEC. 904. STANDARDS OF CARE RELATING TO MUSCULAR 
DYSTROPHY. 
‘‘The Director shall— 
‘‘(1) evaluate the available scientific evidence 
for the appropriate medical or patient organizations 
for purposes of the development and issuance of an 
initial set of care considerations for Duchenne-Beck-
er muscular dystrophy and provide ongoing review 
and updates where appropriate; and 
‘‘(2) replicate the same methodology used to de-
velop the Duchenne-Becker muscular dystrophy care 
considerations developed under paragraph (1) as a 
model for other muscular dystrophies.’’. 
Æ 
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