Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001
PL 107-84 (H.R.717), enacted 12/18/2001

PUBLIC LAW 107-84—DEC. 18, 2001 
115 STAT. 823 
Public Law 107-84 
107th Congress 
An Act 
To amend the Public Health Service Act to provide for research with respect to 
various forms of muscular dystrophy, including Ducheime» Becker, limb girdle, 
coi^enital, facioscapulohumeral, myotonic, oculopharyngeal, distal, and Emery-
Dreifuss muscular dystrophies. 
Be it enacted by the Senate and House of Representatives 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 Amendments of 2001", or the 
"MD-CARE Act". 
SEC. 2. FINDINGS. 
Congress makes the following findings: 
(1) Of the childhood muscular dystrophies, Duchenne Mus-
cular Dystrophy (DMD) is the world's most common and cata-
strophic form of genetic childhood disease, and is characterized 
by a rapidly progressive muscle we£ikness that almost always 
results in death, usually by 20 years of age. 
(2) Duchenne muscular dystrophy is genetically inherited, 
and mothers are the carriers in approximately 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 wiU 
have Duchenne muscular dystrophy, and a 50 percent chance 
per birth that her femede offspring will be carriers. 
(4) Duchenne is the most common lethal genetic disorder 
of childhood worldwide, affecting approximately 1 in every 3,500 
boys worldwide. 
(5) Children with muscular dystrophy exhibit extreme 
sjrmptoms of weakness, delay in walking, waddling gait, dif-
ficulty in climbing stairs, and progressive mobility problems 
often in combination with muscle hypertrophy. 
(6) Other forms of muscular dystrophy affecting children 
and 
adults 
include 
Becker, 
limb 
girdle, 
congenital, 
facioscapulohumeral, myotonic, oculopharyngeal, distal, and 
Emery-Dreifuss muscular dystrophies. 
(7) Myotonic muscular dystrophy (also known as Steinert's 
disease £ind dystrophia myotonica) is the second most prominent 
form of muscul£ir dystrophy and ihe type most commonly found 
in adults. Unlike any of the other muscular dystrophies, the 
muscle weakness is accompsinied by myotonia (delayed relax-
ation of muscles after contraction) £ind by a variety of abnor-
malities in addition to those of muscle. 
Dec. 18, 2001 
[H.R. 717] 
Musculai: 
Dystrophy 
Community 
Assistance, 
Research and 
Education 
Amendments of 
2001. 
42 use 201 note. 
42 use 247b-18 
note. 
115 STAT. 824 
PUBLIC LAW 107-84—DEC. 18, 2001 
(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 £uid sever loss of skeletal muscle gradually 
bringing weakness and reduced mobility. Many persons witn 
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 musculeu" dystrophies, though distinct 
in progressivity Euid severity of S3miptoms, have a devastating 
impact on tens of thousands of children and adults throughout 
; 
the United States Euid worldwide £uid impose severe physiced 
and economic burdens on those affected. 
(11) Muscular dystrophies have a significEUit impact on 
quality of life—not only for the individual who experiences 
its pzdnful S3rmptoms and resulting disability, but also for family 
members and caregivers. 
(12) Development of therapies for these disorders, while 
resdistic with recent advances in reseeu-ch, is likely to require 
costly investments and infrastructure to support gene and other 
therapies. 
(13) There is a shortage of qusdified researchers in the 
field of neuromuscular research. 
(14) Many family physicieuis and health care professionals 
lack the knowledge and resources to detect £uid 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 
cKnically relevant findings in muscular dystrophy rese2u*ch from 
basic science to applied work. 
(16) Educating the public and health care community 
throughout the country about this devastating disease is of 
pareunount importance and is in every respect in the public 
interest eind to the benefit of sJl 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: 
42 use 283g. 
"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 £aid 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 
PUBLIC LAW 107-84—DEC. 18, 2001 
115 STAT. 825 
dystrophy (referred to in this section as 'FSHD') and other 
forms of musculgir 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 imder section 
. 
6oftheMD-CAREAct. 
"(3) ALLOCATIONS BY DIRECTOR OF NIH.—The Director of 
NIH shall allocate the sunoimts appropriated to carry out this 
c;,: 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 Grants. 
and contracts under subsection (a)(1) to public or nonprofit Contracts. 
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 vEuious forms 
of muscular dystrophy. 
"(2) RESEARCH.—Each center under paragraph (1) shall 
supplement but not replace the estabUshment of a comprehen-
sive research portfolio in all the muscular dystrophies. As a 
whole, the centers shaU 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 xmder paragraph (1) and 
ensiu-e 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 fi-om 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 tmder 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 
yeEU-s if the operations of such center have been reviewed 
by an appropriate technical and scientific peer review group 
estabUshed by the Director of NIH and if such group has 
recommended to the Director that such period shoidd 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 reseeirch. The program shall be carried out in 
accordance with accepted scientific and medical stsuidards for the 
donation, collection, and preservation of such samples. 
"(d) COORDINATING COMMITTEE.— 
115 STAT. 826 
PUBLIC LAW 107-84—DEC. 18, 2001 
Establishment. 
Applicability. 
Deadline. 
"(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) % of such members shall represent governmental 
agencies, including the directors or their designees of each 
of the nationsil research institutes involved in research 
with respect to muscular dystrophy and representatives 
of all other Federal departments £uid 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 Depgirtment 
of Education; and 
f 
"(B) Va of such members shall be public members, 
including a broad cross section of persons affected with 
muscular dystrophies including parents or legal guardians, 
affected in<fividuals, researchers, and clinicians. 
Members appointed under subpeiragraph (B) shedl 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 Secreteiry, the Assistant Sec-
retgiry for Hesilth, 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 £md 
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 Humsui Services. 
"(B) The Coordinating Committee shall meet as appro-
priate as determined by the Secretary, in consultation with 
the chair. 
"(e) PLAN FOR H H S 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 resecirch 
institutes and shall periodically review and revise the plan. 
The plan shall— 
PUBLIC LAW 107-84—DEC. 18, 2001 
115 STAT. 827 
"(A) provide for a broad range of research and edu-
cation activities relating to biomedical, epidemiological, 
psychosocial, and rehabihtative issues, including studies 
2 
of the impact of such diseases in rural and underserved 
4JU 
communities; 
"(B) identify priorities among the progreims and activi-
-" 
ties of the National Institutes of Health regarding such 
f»ff; 
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 rese£irch concerning the etiology and genetic 
' 
links of the disease £uid 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 professioneds £uid the public. 
"(0 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, £uid 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 (eXD (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 Heedth 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 
canying out this section, there are authorized to be appropriated 
such sums as may be necessary for each of fiscal yeeirs 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.". 
115 STAT. 828 
PUBLIC LAW 107-84—DEC. 18, 2001 
. 
. 
SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVmES OF CENTERS 
FOR DISEASE CONTROL AND PREVENTION WITH 
RESPECT TO EPIDEMIOLOGICAL RESEARCH ON MUS-
CULAR DYSTROPHY. 
Part B of title III of the Public Health Service Act (42 U.S.C. 
243 et seq.) is amended by inserting after section 317P the following: 
42 use 24'7b-18. <'SEC. 317Q. SURVEILLANCE AND RESEARCH REGARDING MUSCULAR 
DYSTROPHY. 
"(a) IN GENERAL.—The Secretary, acting through the Director 
of the Centers for Disease Control and Prevention, may award 
grants and cooperative agreements to public or nonprofit private 
entities (including health departments of States and political sub-
divisions of States, and including universities £uid other educational 
entities) for the collection, analysis, and reporting of data on 
Duchenne and other forms of muscular dystrophy. In msiking such 
awards, the Secretary may provide direct technical assistance in 
lieu of cash. 
"(b) 
NATIONAL MUSCULAR DYSTROPHY EPIDEMIOLOGY 
PRO-
GRAM.—The Secretary, acting through the Director of the Centers 
for Disease Control and Prevention, may award grants to public 
or nonprofit private entities (including health departments of States 
and political subdivisions of States, and including universities and 
other educational entities) for the purpose of canying out 
epidemiologicsd activities regarding Duchenne and other forms of 
muscular dystrophies, including collecting and analyzing informa-
tion on the nimiber, incidence, correlates, and symptoms of cases. 
In carrjdng out the preceding sentence, the Secretary shall provide 
for a national siu-veillance program. In making awards under this 
subsection, the Secretary may provide direct technical assistance 
in lieu of cash. 
"(c) COORDINATION WiTH CENTERS OF EXCELLENCE.—The Sec-
retary shsdl ensure that epidemiological information under sub-
sections (a) and (b) is made avEulable to centers of excellence sup-
ported under section 404E(b) by the Director of the National 
Institutes of Health. 
"(d) AUTHORIZATION OF APPROPRIATIONS.—^There are authorized 
to be appropriated such simis as may be necessary to carry out 
this section.". 
42 use 247b-19. 
SEC. 5. INFORMATION AND EDUCATION. 
(a) IN GENERAL.—^The Secretary of Health £ind Human Services 
(referred to in this Act as the "Secretary") shall establish and 
implement a program to provide information and education on 
musculeir dystrophy to health professionals and the general public, 
including information and education on advsuices in the diagnosis 
and treatment of muscular dystrophy and training Euid continuing 
education through programs for scientists, physicians, medical stu-
dents, and other heedth professionals who provide care for patients 
with muscular dystrophy. 
(b) STIPENDS.—llie Secretary may use amounts made available 
under this section provides 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. 
PUBLIC LAW 107-84—DEC. 18, 2001 
115 STAT. 829 
SEC. 6. REPORT TO CONGRESS. 
42 USC 247b-18 
Not later than January 1, 2003, and each January 1 thereafter. Deadline, 
the Secretary shall prepare and submit to the appropriate commit-
tees of Congress, a report concerning the implementation of this 
Act and the amendments made by this Act. 
SEC. 7. STUDY ON THE USE OF CENTERS OF EXCELLENCE AT THE 
NATIONAL INSTITUTES OF HEALTH. 
42 USC 281 note. 
Deadline. 
Contradis. 
(a) REVIEW.—Not later than 60 days after the date of enactment 
of this Act, the Secretary of Headth and Human Services shall 
enter into a contract with the Institute of Medicine for the purpose 
of conducting a study and making recommendations on the impact 
of, need for, and other issues associated with Centers of Excellence 
at the National Institutes of Health. 
(b) AREAS OF REVIEW.—In conducting the study under sub-
section (a), the Institute of Medicine shall at a minimum consider 
the following: 
(1) The current areas of research incorporating Centers 
of Excellence (which shall include a description of such £u*eas) 
and the relationship of this form of funding mechanism to 
other forms of funding for research grants, including investi-
; gator initiated research, contracts and other types of reseeu-ch 
support awards. 
(2) The distinctive aspects of Centers of Excellence, 
including the additional knowledge that may be expected to 
be gained through Centers of Excellence as compared to other 
forms of grant or contract mechanisms. 
(3) The costs associated with establishing and mmntaining 
Centers of Excellence, Euid the record of scholarship and 
trsuning resulting from such Centers. The research and training 
contributions of Centers should be assessed on their own merits 
and in comparison with other forms of research support. 
(4) Specific areas of research in which Centers of Excellence 
may be useful, needed, or imderused, as well as areas of 
research in which Centers of Excellence may not be helpful. 
(5) Criteria that may be appUed in determining when Cen-
ters of Excellence are an appropriate and cost-effective research 
investment and conditions that should be present in order 
to consider the establishment of Centers of Excellence. 
(6) Alternative research models that may accomplish 
results similar to or greater than Centers of Excellence. 
(c) REPORT.—Not later than 1 year after the date on which Deadline, 
the contract is entered into under subsection (a), the Institute 
of Medicine shall complete the study under such subsection and 
submit a report to the Secretary of Headth and Human Services 
115 STAT. 830 
PUBLIC LAW 107-84—DEC. 18, 2001 
and the appropriate committees of Congress that contains the 
results of such study. 
Approved December 18, 2001. 
LEGISLATIVE fflSTORY—H.R. 717: 
HOUSE REPORTS: No. 107-195 (Comm. on Energy and Commerce). 
CONGRESSIONAL RECORD, Vol. 147 (2001): 
Sept. 24, considered and passed House. 
Nov. 15, considered and passed Senate, amended. 
Nov. 29, House concurred in Senate amendment.