107TH CONGRESS
1ST SESSION
H. R. 717
AN ACT
To amend the Public Health Service Act to provide for
research with respect to various forms of muscular dys-
trophy, including Duchenne, Becker, limb girdle, con-
genital, facioscapulohumeral, myotonic, oculopharyngeal,
distal, and Emery-Dreifuss 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 ‘‘Muscular Dystrophy
Community Assistance, Research and Education Amend-
ments of 2001’’, or the ‘‘MD–CARE Act’’.
SEC. 2. FINDINGS.
Congress makes the following findings:
(1) Of the childhood muscular dystrophies,
Duchenne Muscular Dystrophy (DMD) is the
world’s most common and catastrophic form of ge-
netic childhood disease, and is characterized by a
rapidly progressive muscle weakness that almost al-
ways results in death, usually by 20 years of age.
(2) Duchenne muscular dystrophy is genetically
inherited, and mothers are the carriers in approxi-
mately 70 percent of all cases.
(3) If a female is a carrier of the dystrophin
gene, there is a 50 percent chance per birth that her
male offspring will have Duchenne muscular dys-
trophy, and a 50 percent chance per birth that her
female offspring will be carriers.
(4) Duchenne is the most common lethal ge-
netic disorder of childhood worldwide, affecting ap-
proximately 1 in every 3,500 boys worldwide.
(5) Children with muscular dystrophy exhibit
extreme symptoms of weakness, delay in walking,
waddling gait, difficulty in climbing stairs, and pro-
gressive mobility problems often in combination with
muscle hypertrophy.
(6) Other forms of muscular dystrophy affect-
ing children and adults include Becker, limb girdle,
congenital, 
facioscapulohumeral, 
myotonic,
oculopharyngeal, distal, and Emery-Dreifuss mus-
cular dystrophies.
(7) Myotonic muscular dystrophy (also known
as Steinert’s disease and dystrophia myotonica) is
the second most prominent form of muscular dys-
trophy and the type most commonly found in adults.
Unlike any of the other muscular dystrophies, the
muscle weakness is accompanied by myotonia (de-
layed relaxation of muscles after contraction) and by
a variety of abnormalities in addition to those of
muscle.
(8) Facioscapulohumeral muscular dystrophy
(referred to in this section as ‘‘FSHD’’) is a neuro-
muscular disorder that is inherited genetically and
has an estimated frequency of 1 in 20,000. FSHD,
affecting between 15,000 to 40,000 persons, causes
a progressive and sever loss of skeletal muscle
gradually bringing weakness and reduced mobility.
Many persons with FSHD become severely phys-
ically disabled and spend many decades in a wheel-
chair.
(9) FSHD is regarded as a novel genetic phe-
nomenon resulting from a crossover of subtelomeric
DNA and may be the only human disease caused by
a deletion-mutation.
(10) Each of the muscular dystrophies, though
distinct in progressivity and severity of symptoms,
have a devastating impact on tens of thousands of
children and adults throughout the United States
and worldwide and impose severe physical and eco-
nomic burdens on those affected.
(11) Muscular dystrophies 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 care-
givers.
(12) Development of therapies for these dis-
orders, while realistic with recent advances in re-
search, is likely to require costly investments and in-
frastructure to support gene and other therapies.
(13) There is a shortage of qualified research-
ers in the field of neuromuscular research.
(14) Many family physicians and health care
professionals lack the knowledge and resources to
detect and properly diagnose the disease as early as
possible, thus exacerbating the progressiveness of
symptoms 
in 
cases 
that 
go 
undetected 
or
misdiagnosed.
(15) There is a need for efficient mechanisms
to translate clinically relevant findings in muscular
dystrophy research from basic science to applied
work.
(16) Educating the public and health care com-
munity throughout the country about this dev-
astating disease is of paramount importance and is
in every respect in the public interest and to the
benefit of all communities.
SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINA-
TION OF ACTIVITIES OF NATIONAL INSTI-
TUTES OF HEALTH WITH RESPECT TO RE-
SEARCH ON MUSCULAR DYSTROPHY.
Part A of title IV of the Public Health Service Act
(42 U.S.C. 281 et seq.) is amended by adding at the end
the following:
‘‘SEC. 404E. MUSCULAR DYSTROPHY; INITIATIVE THROUGH
DIRECTOR OF NATIONAL INSTITUTES OF
HEALTH.
‘‘(a) EXPANSION, INTENSIFICATION, AND COORDINA-
TION OF ACTIVITIES.—
‘‘(1) IN GENERAL.—The Director of NIH, in
coordination with the Directors of the National In-
stitute of Neurological Disorders and Stroke, the
National Institute of Arthritis and Muscoskeletal
and Skin Diseases, the National Institute of Child
Health and Human Development, and the other na-
tional research institutes as appropriate, shall ex-
pand and intensify programs of such Institutes with
respect to research and related activities concerning
various forms of muscular dystrophy, including
Duchenne, myotonic, facioscapulohumeral muscular
dystrophy (referred to in this section as ‘FSHD’)
and other forms of muscular dystrophy.
‘‘(2) COORDINATION.—The Directors referred
to in paragraph (1) shall jointly coordinate the pro-
grams referred to in such paragraph and consult
with the Muscular Dystrophy Interagency Coordi-
nating Committee established under section 6 of the
MD–CARE Act.
‘‘(3) ALLOCATIONS BY DIRECTOR OF NIH.—The
Director of NIH shall allocate the amounts appro-