II
Calendar No. 208
107TH CONGRESS
1ST SESSION
H. R. 717
IN THE SENATE OF THE UNITED STATES
SEPTEMBER 25, 2001
Received; read twice and referred to the Committee on Health, Education,
Labor, and Pensions
OCTOBER 30, 2001
Reported by Mr. KENNEDY, with an amendment
[Insert the part printed in italic]
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-
priated to carry out this section for each fiscal year
among the national research institutes referred to in
paragraph (1).
‘‘(b) CENTERS OF EXCELLENCE.—
‘‘(1) IN GENERAL.—The Director of NIH shall
award grants and contracts under subsection (a)(1)
to public or nonprofit private entities to pay all or
part of the cost of planning, establishing, improving,
and providing basic operating support for centers of
excellence regarding research on various forms of
muscular dystrophy.
‘‘(2) RESEARCH.—Each center under para-
graph (1) shall supplement but not replace the es-
tablishment of a comprehensive research portfolio in
all the muscular dystrophies. As a whole, the centers
shall conduct basic and clinical research in all forms
of muscular dystrophy including early detection, di-
agnosis, prevention, and treatment, including the
fields of muscle biology, genetics, noninvasive imag-
ing, genetics, pharmacological and other therapies.
‘‘(3) COORDINATION OF CENTERS; REPORTS.—
The Director of NIH—
‘‘(A) shall, as appropriate, provide for the
coordination of information among centers
under paragraph (1) and ensure regular com-
munication between such centers; and
‘‘(B) shall require the periodic preparation
of reports on the activities of the centers and
the submission of the reports to the Director.
‘‘(4) ORGANIZATION OF CENTERS.—Each cen-
ter under paragraph (1) shall use the facilities of a
single institution, or be formed from a consortium of
cooperating institutions, meeting such requirements
as may be prescribed by the Director of NIH.
‘‘(5) DURATION OF SUPPORT.—Support for a
center established under paragraph (1) may be pro-
vided under this section for a period of not to exceed
5 years. Such period may be extended for 1 or more
additional periods not exceeding 5 years if the oper-
ations of such center have been reviewed by an ap-
propriate technical and scientific peer review group
established by the Director of NIH and if such
group has recommended to the Director that such
period should be extended.
‘‘(c) FACILITATION OF RESEARCH.—The Director of
NIH shall provide for a program under subsection (a)(1)
under which samples of tissues and genetic materials that
are of use in research on muscular dystrophy are donated,
collected, preserved, and made available for such research.
The program shall be carried out in accordance with ac-
cepted scientific and medical standards for the donation,
collection, and preservation of such samples.
‘‘(d) COORDINATING COMMITTEE.—
‘‘(1) IN GENERAL.—The Secretary shall estab-
lish the Muscular Dystrophy Coordinating Com-
mittee (referred to in this section as the ‘Coordi-
nating Committee’) to coordinate activities across
the National Institutes and with other Federal
health programs and activities relating to the var-
ious forms of muscular dystrophy.
‘‘(2) COMPOSITION.—The Coordinating Com-
mittee shall consist of not more than 15 members to
be appointed by the Secretary, of which—
‘‘(A) 2⁄3 of such members shall represent
governmental agencies, including the directors
or their designees of each of the national re-
search institutes involved in research with re-
spect to muscular dystrophy and representatives
of all other Federal departments and agencies
whose programs involve health functions or re-
sponsibilities relevant to such diseases, includ-
ing the Centers for Disease Control and Pre-
vention, the Health Resources and Services Ad-
ministration and the Food and Drug Adminis-
tration and representatives of other govern-
mental agencies that serve children with mus-
cular dystrophy, such as the Department of
Education; and
‘‘(B) 1⁄3 of such members shall be public
members, including a broad cross section of
persons affected with muscular dystrophies in-
cluding parents or legal guardians, affected in-
dividuals, researchers, and clinicians.
Members appointed under subparagraph (B) shall
serve for a term of 3 years, and may serve for an
unlimited number of terms if reappointed.
‘‘(3) CHAIR.—
‘‘(A) IN GENERAL.—With respect to mus-
cular dystrophy, the Chair of the Coordinating
Committee shall serve as the principal advisor
to the Secretary, the Assistant Secretary for
Health, and the Director of NIH, and shall pro-
vide advice to the Director of the Centers for
Disease Control and Prevention, the Commis-
sioner of Food and Drugs, and to the heads of
other relevant agencies. The Coordinating Com-
mittee shall select the Chair for a term not to
exceed 2 years.
‘‘(B) APPOINTMENT.—The Chair of the
Committee shall be appointed by and be directly
responsible to the Secretary.
‘‘(4) ADMINISTRATIVE
SUPPORT; TERMS
OF
SERVICE; OTHER PROVISIONS.—The following shall
apply with respect to the Coordinating Committee:
‘‘(A) The Coordinating Committee shall re-
ceive necessary and appropriate administrative
support from the Department of Health and
Human Services.
‘‘(B) The Coordinating Committee shall
meet as appropriate as determined by the Sec-
retary, in consultation with the chair.
‘‘(e) PLAN FOR HHS ACTIVITIES.—
‘‘(1) IN GENERAL.—Not later than 1 year after
the date of enactment of this section, the Coordi-
nating Committee shall develop a plan for con-
ducting and supporting research and education on
muscular dystrophy through the national research
institutes and shall periodically review and revise the
plan. The plan shall—
‘‘(A) provide for a broad range of research
and education activities relating to biomedical,
epidemiological, psychosocial, and rehabilitative
issues, including studies of the impact of such
diseases in rural and underserved communities;
‘‘(B) identify priorities among the pro-
grams and activities of the National Institutes
of Health regarding such diseases; and
‘‘(C) reflect input from a broad range of
scientists, patients, and advocacy groups.
‘‘(2) CERTAIN ELEMENTS OF PLAN.—The plan
under paragraph (1) shall, with respect to each form
of muscular dystrophy, provide for the following as
appropriate:
‘‘(A) Research to determine the reasons
underlying the incidence and prevalence of var-
ious forms of muscular dystrophy.
‘‘(B) Basic research concerning the eti-
ology and genetic links of the disease and po-
tential causes of mutations.
‘‘(C) The development of improved screen-
ing techniques.
‘‘(D) Basic and clinical research for the
development and evaluation of new treatments,
including new biological agents.
‘‘(E) Information and education programs
for health care professionals and the public.
‘‘(f) REPORTS
TO CONGRESS.—The Coordinating
Committee shall biennially submit to the Committee on
Energy and Commerce of the House of Representatives,
and the Committee on Health, Education, Labor, and
Pensions of the Senate, a report that describes the re-
search, education, and other activities on muscular dys-
trophy being conducted or supported through the Depart-
ment of Health and Human Services. Each such report
shall include the following:
‘‘(1) The plan under subsection (e)(1) (or revi-
sions to the plan, as the case may be).
‘‘(2) Provisions specifying the amounts ex-
pended by the Department of Health and Human
Services with respect to various forms of muscular
dystrophy, including Duchenne, myotonic, FSHD
and other forms of muscular dystrophy.
‘‘(3) Provisions identifying particular projects
or types of projects that should in the future be con-
sidered by the national research institutes or other
entities in the field of research on all muscular dys-
trophies.
‘‘(g) PUBLIC INPUT.—The Secretary shall, under
subsection (a)(1), provide for a means through which the
public can obtain information on the existing and planned
programs and activities of the Department of Health and
Human Services with respect to various forms of muscular
dystrophy and through which the Secretary can receive
comments from the public regarding such programs and
activities.
‘‘(h) AUTHORIZATION OF APPROPRIATIONS.—For the
purpose of carrying out this section, there are authorized
to be appropriated such sums as may be necessary for
each of fiscal years 2002 through 2006. The authorization
of appropriations established in the preceding sentence is