I
107TH CONGRESS
1ST SESSION
H. R. 717
To amend the Public Health Service Act to provide for research and services
with respect to Duchenne muscular dystrophy.
IN THE HOUSE OF REPRESENTATIVES
FEBRUARY 14, 2001
Mr. WICKER (for himself, Mr. PETERSON of Minnesota, Mr. GREENWOOD, Mr.
TANNER, Mr. EHRLICH, Mr. CRAMER, Mr. GORDON, Mrs. EMERSON, Mr.
RILEY, Mr. BRYANT, Mr. FORD, Mr. FOLEY, Ms. HOOLEY of Oregon,
Mr. KING, Mr. HOBSON, Mr. PICKERING, Mr. CHAMBLISS, Mr. EHLERS,
Mr. TOWNS, Mr. MCGOVERN, Mr. LATOURETTE, Mr. DOOLITTLE, Mr.
WATTS of Oklahoma, Ms. GRANGER, Mr. BLUMENAUER, Mr. MURTHA,
Mr. OLVER, Mr. BOEHLERT, Mr. GOODLATTE, Mr. HOLDEN, Mr. WAT-
KINS, Mr. COBLE, Mr. ISAKSON, Mr. LOBIONDO, Mr. MCCRERY, Mr.
KERNS, Mr. GILMAN, Mr. ROHRABACHER, Mr. ISSA, Mr. CALVERT, Mr.
LANGEVIN, Mrs. MEEK of Florida, Mr. HASTINGS of Florida, Ms. BROWN
of Florida, Mr. MILLER of Florida, Mr. OTTER, Mr. WALDEN of Oregon,
Mrs. MYRICK, Mr. LAHOOD, Mr. LIPINSKI, Mr. LEWIS of Kentucky, Mr.
WOLF, Mr. HOSTETTLER, Mr. KINGSTON, Mr. SCARBOROUGH, Mr.
UPTON, Mr. LEACH, Mr. GILLMOR, Mr. WALSH, Mr. QUINN, Mr.
GANSKE, Mr. JONES of North Carolina, Mr. BACHUS, Mr. OXLEY, Mr.
TIAHRT, Mr. WELLER, Mr. MATSUI, Mr. WELDON of Florida, Mr. REY-
NOLDS, Mr. GUTKNECHT, Mr. CHABOT, Mr. HUNTER, Mr. GOODE, Mr.
FLETCHER, Mr. SKELTON, Mr. MORAN of Virginia, Mr. RODRIGUEZ, Mr.
TURNER, Mr. BENTSEN, Mr. ABERCROMBIE, Mr. GONZALEZ, Mr. BILI-
RAKIS, Mr. ARMEY, Mr. MCHUGH, Mr. JENKINS, Mr. BOYD, Mr. PUT-
NAM, Mr. ROGERS of Michigan, Mr. KELLER, Mrs. KELLY, and Mr.
MANZULLO) introduced the following bill; which was referred to the Com-
mittee on Energy and Commerce
A BILL
To amend the Public Health Service Act to provide for
research and services with respect to Duchenne muscular
dystrophy.
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 ‘‘Duchenne Muscular
Dystrophy Childhood Assistance, Research and Education
Amendments of 2001’’, or the ‘‘DMD CARE Act’’.
SEC. 2. FINDINGS.
The Congress finds as follows:
(1) Duchenne is the most common form of mus-
cular dystrophy, and is characterized by a rapidly
progressive muscle weakness that almost always re-
sults 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) Children with Duchenne muscular dys-
trophy exhibit extreme symptoms of weakness, delay
in walking, waddling gait, difficulty in climbing
stairs, and progressive mobility problems often in
combination with muscle hypertrophy.
(5) Duchenne is the most common lethal ge-
netic disorder of childhood worldwide, affecting ap-
proximately one in every 3,500 boys worldwide.
(6) Although the dystrophin gene that causes
DMD was successfully identified and isolated by
medical researchers in 1987, Federal research de-
voted to potential treatment options or a cure since
this initial discovery has been minimal.
(7) Because of the limited Federal support for
medical research specific to this form of muscular
dystrophy, current treatment options are minimal in
efficacy and palliative, aimed at simply managing
the symptoms in an effort to optimize the quality of
life.
(8) Many family physicians and health care pro-
fessionals lack the knowledge and resources to detect
and properly diagnose the disease as early as pos-
sible, thus exacerbating the progressiveness of symp-
toms in cases that go undetected or misdiagnosed.
(9) This disease has a significant impact on
quality of life—not only for the individual who expe-
riences its painful symptoms and resulting disability,
but also for family members and caregivers.
stitute of Neurological Disorders and Stroke, Na-
tional Institute of Arthritis and Muscoskeletal and
Skin Diseases and the National Institute of Child
Health and Human Development, shall expand and
intensify programs of such Institutes with respect to
research and related activities concerning Duchenne
muscular dystrophy.
‘‘(2) COORDINATION.—The Directors referred
to in subsection (a) shall jointly coordinate the pro-
grams referred to in such subsection and consult
with the Muscular Dystrophy Interagency Coordi-
nating Committee.
‘‘(3) ALLOCATIONS
BY
DIRECTOR
OF
NIH.—
With respect to amounts appropriated to carry out
this section for a fiscal year, the Director of NIH
shall allocate the amounts among the national re-
search institutes that are referred to in subsection
(a).
‘‘(b) CENTERS OF EXCELLENCE.—
‘‘(1) IN GENERAL.—The Director shall under
subsection (a)(1) make awards of grants and con-
tracts to public or nonprofit private entities to pay
all or part of the cost of planning, establishing, im-
proving, and providing basic operating support for
centers 
of 
excellence 
regarding 
research 
on
Duchenne muscular dystrophy.
‘‘(2) RESEARCH.—Each center under para-
graph (1) shall conduct basic and clinical research
into Duchenne muscular dystrophy. Such research
should include investigations into the diagnosis,
early detection, prevention, control, and adequate
treatment of Duchenne muscular dystrophy. The
centers, as a group, shall conduct research including
the fields of muscle biology, genetics, non-invasive
imaging, gene and pharmaceutical therapies.
‘‘(3) COORDINATION OF CENTERS; REPORTS.—
The Director shall, as appropriate, provide for the
coordination of information among centers under
paragraph (1) and ensure regular communication
between such centers, and may 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.
‘‘(5) NUMBER OF CENTERS; DURATION OF SUP-
PORT.—
‘‘(A) IN
GENERAL.—The Director shall
provide for the establishment of not less than
three centers under paragraph (1).
‘‘(B) DURATION.—Support for a center es-
tablished under paragraph (1) may be provided
under this section for a period of not to exceed
5 years. Such period may be extended for one
or more additional periods not exceeding 5
years if the operations of such center have been
reviewed by an appropriate technical and sci-
entific peer review group established by the Di-
rector and if such group has recommended to
the Director that such period should be ex-
tended.
‘‘(c) FACILITATION
OF RESEARCH.—The Director
shall under subsection (a)(1) provide for a program under
which samples of tissues and genetic materials that are
of use in research on Duchenne muscular dystrophy are
donated, collected, preserved, and made available for such
research. The program shall be carried out in accordance
with accepted scientific and medical standards for the do-
nation, 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 such dis-
eases.
‘‘(2) COMPOSITION.—The Coordinating Com-
mittee shall be composed of the directors or their
designees of each of the national research institutes
involved in research with respect to Duchenne mus-
cular dystrophy and representatives of all other Fed-
eral departments and agencies whose programs in-
volve health functions or responsibilities relevant to
such diseases, including the Centers for Disease
Control and Prevention, the Health Services and Re-
sources Administration and the Food and Drug Ad-
ministration.
‘‘(3) CHAIR.—
‘‘(A) 
IN
GENERAL.—With 
respect 
to
Duchenne muscular dystrophy, the Chair of the
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 other relevant
agencies.
‘‘(B) DIRECTOR
OF
NIH.—The Chair of
the Committee shall be directly responsible to
the Director of NIH.
‘‘(e) PLAN FOR NIH ACTIVITIES.—
‘‘(1) IN GENERAL.—Not later than 1 year after
the date of the enactment of this section, the Coordi-
nating Committee shall develop a plan for con-
ducting and supporting research and education on
Duchenne 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,
psychosocial, and rehabilitative issues, including
studies of the impact of such diseases on boys
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 Duchenne
muscular dystrophy, provide for the following as ap-
propriate:
‘‘(A) Research to determine the reasons
underlying the incidence and prevalence of
Duchenne 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.
‘‘(3) IMPLEMENTATION OF PLAN.—The Direc-
tor of NIH shall ensure that programs and activities
of the National Institutes of Health regarding
Duchenne muscular dystrophy are implemented in
accordance with the plan under paragraph (1).
‘‘(f) REPORTS
TO CONGRESS.—The Coordinating
Committee under subsection (b)(1) shall biennially submit
to the Committee on Commerce of the House of Rep-
resentatives, and the Committee on Health, Education,
Labor and Pensions of the Senate, a report that describes
ization of appropriations established in the preceding sen-
tence is in addition to any other authorization of appro-
priations that is available for conducting or supporting
through the National Institutes of Health research and
other activities with respect to Duchenne muscular dys-
trophy.’’.
SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF
CENTERS FOR DISEASE CONTROL AND PRE-
VENTION WITH RESPECT TO EPIDEMIOLOG-
ICAL RESEARCH ON DUCHENNE MUSCULAR
DYSTROPHY.
Part B of title III of the Public Health Service Act
(42 U.S.C. 243 et seq.) is amended by inserting after sec-
tion 317P the following section:
‘‘SURVEILLANCE AND RESEARCH REGARDING DUCHENNE
MUSCULAR DYSTROPHY
‘‘SEC. 317Q. (a) IN GENERAL.—The Secretary, act-
ing through the Director of the Centers for Disease Con-
trol and Prevention, may make awards of grants and coop-
erative agreements for the collection, analysis, and report-
ing of data on Duchenne muscular dystrophy. An entity
may receive such an award only if the entity is a public
or nonprofit private entity (including health departments
of States and subdivisions of States, and including univer-
sities and other educational entities). In making such
awards, the Secretary may provide direct technical assist-
ance in lieu of cash.
‘‘(b) NATIONAL DUCHENNE MUSCULAR DYSTROPHY
SURVEILLANCE PROGRAM.—
‘‘(1) IN GENERAL.—The Secretary of Health
and Human Services (in this section referred to as
the ‘Secretary’), acting through the Director of the
Centers for Disease Control and Prevention, may
make awards of grants and cooperative agreements
for the collection, analysis, and reporting of data on
Duchenne muscular dystrophy. In making such
awards, the Secretary may provide direct technical
assistance in lieu of cash.
‘‘(2) ELIGIBILITY.—To be eligible to receive an
award under paragraph (1) an entity shall be a pub-
lic or nonprofit private entity (including health de-
partments of States and political subdivisions of
States, and including universities and other edu-
cational entities).
‘‘(c) CENTERS OF EXCELLENCE IN DUCHENNE AND
RELATED MUSCULAR DYSTROPHIES EPIDEMIOLOGY.—
‘‘(1) IN
GENERAL.—The Secretary, acting
through the Director of the Centers for Disease
Control and Prevention, shall establish not less than
three regional centers of excellence in Duchenne
muscular dystrophy epidemiology for the purpose of
collecting and analyzing information on the number,
incidence, correlates, and symptoms of Duchenne
and related muscular dystrophies.
‘‘(2) RECIPIENTS OF AWARDS FOR ESTABLISH-
MENT OF CENTERS.—Centers under paragraph (1)
shall be established and operated through the award-
ing of grants or cooperative agreements to public or
nonprofit private entities that conduct research, in-
cluding health departments of States and political
subdivisions of States, and including universities and
other educational entities.
‘‘(3) CERTAIN REQUIREMENTS.—An award for
a center under paragraph (1) may be made only if
the entity involved submits to the Secretary an ap-
plication containing such agreements and informa-
tion as the Secretary may require, including an
agreement that the center involved will operate in
accordance with the following:
‘‘(A) The center will collect, analyze, and
report Duchenne muscular dystrophy data ac-
cording to guidelines prescribed by the Director,
after consultation with relevant State and local
public health officials, private sector research-
ers, and advocates for those with Duchenne
muscular dystrophy.
‘‘(B) The center will assist with the devel-
opment and coordination of State Duchenne
and related muscular dystrophy surveillance ef-
forts within a region.
‘‘(C) The center will identify eligible cases
and controls through its surveillance systems
and conduct research into factors which may
cause Duchenne muscular dystrophy.
‘‘(D) The center will develop or extend an
area of special research expertise (including ge-
netics, immunology, and other relevant research
specialty areas).
‘‘(d) DEFINITION.—In this title, the term ‘State’
means each of the several States, the District of Columbia,
the Commonwealth of Puerto Rico, American Samoa,
Guam, the Commonwealth of the Northern Mariana Is-
lands, the Virgin Islands, and the Trust Territory of the
Pacific Islands.
‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—There
are authorized to be appropriated such sums as may be
necessary to carry out this section.’’.
SEC. 5. INFORMATION AND EDUCATION.
(a) IN GENERAL.—The Secretary shall establish and
implement a program to provide information and edu-
cation on Duchenne muscular dystrophy to health profes-
sionals and the general public, including information and
education on advances in the diagnosis and treatment of
Duchenne muscular dystrophy and training and con-
tinuing education through programs for scientists, physi-
cians, and other health professionals who provide care for
patients with Duchenne muscular dystrophy.
(b) STIPENDS.—The Secretary may use amounts
made available under this section to provide stipends for
health professionals who are enrolled in training programs
under this section.
(c) AUTHORIZATION
OF APPROPRIATIONS.—There
are authorized to be appropriated such sums as may be
necessary to carry out this section.
SEC. 6. INTER-AGENCY MUSCULAR DYSTROPHY COORDI-
NATING COMMITTEE.
(a) ESTABLISHMENT.—The Secretary shall establish
a committee to be known as the ‘‘Muscular Dystrophy Co-
ordinating Committee’’ (in this section referred to as the
‘‘Committee’’) to coordinate all efforts within the Depart-
ment of Health and Human Services concerning Duchenne
muscular dystrophy, including activities carried out
through the National Institutes of Health and the Centers
for Disease Control and Prevention under this Act (and
the amendments made by this Act).
(b) MEMBERSHIP.—
(1) IN
GENERAL.—The Committee shall be
composed of the Directors of such national research
institutes, of the Centers for Disease Control and
Prevention, and of such other agencies and such
other officials as the Secretary determines appro-
priate.
(2) ADDITIONAL MEMBERS.—If determined ap-
propriate by the Secretary, the Secretary may ap-
point to the Committee—
(A) parents or legal guardians of individ-
uals with Duchenne muscular dystrophy; and
(B) representatives of other governmental
agencies that serve children with Duchenne
muscular dystrophy such as the Department of
Education.
(c) ADMINISTRATIVE SUPPORT; TERMS OF SERVICE;
OTHER PROVISIONS.—The following shall apply with re-
spect to the Committee:
(1) The Committee shall receive necessary and
appropriate administrative support from the Depart-
ment of Health and Human Services.
(2) Members of the Committee appointed under
subsection (b)(2)(A) shall serve for a term of 3
years, and may serve for an unlimited number of
terms if reappointed.
(3) The Committee shall meet not less than two
times each year.
SEC. 7. REPORT TO CONGRESS.
Not later than January 1, 2003, and each January
1 thereafter, the Secretary shall prepare and submit to
the appropriate committees of Congress, a report con-
cerning the implementation of this title and the amend-
ments made by this title.
SEC. 8. DEFINITION.
For purposes of this Act, the term ‘‘Secretary’’
means the Secretary of Health and Human Services.
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