H. R. 717—2
(8) Facioscapulohumeral muscular dystrophy (referred to
in this section as ‘‘FSHD’’) is a neuromuscular 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 physically disabled and spend many
decades in a wheelchair.
(9) FSHD is regarded as a novel genetic phenomenon
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 economic 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 caregivers.
(12) Development of therapies for these disorders, while
realistic with recent advances in research, is likely to require
costly investments and infrastructure to support gene and other
therapies.
(13) There is a shortage of qualified researchers 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 community
throughout the country about this devastating 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 COORDINATION OF
ACTIVITIES OF NATIONAL INSTITUTES OF HEALTH WITH
RESPECT TO RESEARCH 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
COORDINATION
OF
ACTIVITIES.—
‘‘(1) IN GENERAL.—The Director of NIH, in coordination
with the Directors of the National Institute 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 national
research institutes as appropriate, shall expand and intensify
programs of such Institutes with respect to research and related
activities concerning various forms of muscular dystrophy,
including Duchenne, myotonic, facioscapulohumeral muscular
H. R. 717—3
dystrophy (referred to in this section as ‘FSHD’) and other
forms of muscular dystrophy.
‘‘(2) COORDINATION.—The Directors referred to in para-
graph (1) shall jointly coordinate the programs referred to
in such paragraph and consult with the Muscular Dystrophy
Interagency Coordinating Committee established under section
6 of the MD–CARE Act.
‘‘(3) ALLOCATIONS BY DIRECTOR OF NIH.—The Director of
NIH shall allocate the amounts appropriated 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 paragraph (1) shall
supplement but not replace the establishment of a comprehen-
sive 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,
diagnosis, prevention, and treatment, including the fields of
muscle biology, genetics, noninvasive imaging, 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 communication 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 center under para-
graph (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 estab-
lished under paragraph (1) may be provided 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 operations of such center have been reviewed
by an appropriate 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 accepted scientific and medical standards for the
donation, collection, and preservation of such samples.
‘‘(d) COORDINATING COMMITTEE.—
H. R. 717—4
‘‘(1) IN GENERAL.—The Secretary shall establish the Mus-
cular Dystrophy Coordinating Committee (referred to in this
section as the ‘Coordinating Committee’) to coordinate activities
across the National Institutes and with other Federal health
programs and activities relating to the various forms of mus-
cular dystrophy.
‘‘(2) COMPOSITION.—The Coordinating Committee shall con-
sist 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 research institutes involved in research
with respect to muscular dystrophy and representatives
of all other Federal departments and agencies whose pro-
grams involve health functions or responsibilities relevant
to such diseases, including the Centers for Disease Control
and Prevention, the Health Resources and Services
Administration and the Food and Drug Administration and
representatives of other governmental agencies that serve
children with muscular 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 including parents or legal guardians,
affected individuals, 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 muscular dystrophy,
the Chair of the Coordinating Committee shall serve as
the principal advisor to the Secretary, the Assistant Sec-
retary for Health, and the Director of NIH, and shall pro-
vide advice to the Director of the Centers for Disease
Control and Prevention, the Commissioner of Food and
Drugs, and to the heads of other relevant agencies. The
Coordinating Committee 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 Sec-
retary.
‘‘(4) ADMINISTRATIVE SUPPORT; TERMS OF SERVICE; OTHER
PROVISIONS.—The following shall apply with respect to the
Coordinating Committee:
‘‘(A) The Coordinating Committee shall receive nec-
essary and appropriate administrative support from the
Department of Health and Human Services.
‘‘(B) The Coordinating Committee shall meet as appro-
priate as determined by the Secretary, 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 Coordinating Committee shall
develop a plan for conducting and supporting research and
education on muscular dystrophy through the national research
institutes and shall periodically review and revise the plan.
The plan shall—
H. R. 717—5
‘‘(A) provide for a broad range of research and edu-
cation 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 programs and activi-
ties 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 para-
graph (1) shall, with respect to each form of muscular dys-
trophy, provide for the following as appropriate:
‘‘(A) Research to determine the reasons underlying the
incidence and prevalence of various forms of muscular dys-
trophy.
‘‘(B) Basic research concerning the etiology and genetic
links of the disease and potential causes of mutations.
‘‘(C) The development of improved screening tech-
niques.
‘‘(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, Edu-
cation, Labor, and Pensions of the Senate, a report that describes
the research, education, and other activities on muscular dystrophy
being conducted or supported through the Department of Health
and Human Services. Each such report shall include the following:
‘‘(1) The plan under subsection (e)(1) (or revisions to the
plan, as the case may be).
‘‘(2) Provisions specifying the amounts expended 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 considered by the national
research institutes or other entities in the field of research
on all muscular dystrophies.
‘‘(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 pre-
ceding sentence 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 muscular dystrophy.’’.