II 

110TH CONGRESS 

2D SESSION H. R. 5265 

IN THE SENATE OF THE UNITED STATES 

SEPTEMBER 25 (legislative day, SEPTEMBER 17), 2008 

Received 

AN ACT 

To amend the Public Health Service Act to provide for 

research with respect to various forms of muscular dystrophy, including Becker, congenital, distal, Duchenne, 

Emery-Dreifuss facioscapulohumeral, limb-girdle, 

myotonic, and oculopharyngeal, muscular dystrophies. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 


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1 SECTION 1. SHORT TITLE. 

2 This Act may be cited as the ‘‘Paul D. Wellstone 

3 Muscular Dystrophy Community Assistance, Research, 

4 and Education Amendments of 2008’’. 

5 SEC. 2. FINDINGS. 

6 The Congress finds as follows: 

7 (1) The muscular dystrophies are devastating 

8 diseases that have a significant impact on quality of 

9 life—not only for the individual who experiences its 

10 painful symptoms and resulting disability, but also 

11 for family members and caregivers. 

12 (2) DMD is the most common lethal genetic 

13 disorder of childhood worldwide, affecting approxi14 mately 1 in every 3,500 boys born each year around 

15 the globe. It is characterized by a rapidly progressive 

16 muscle weakness that almost always results in death 

17 from respiratory or cardiac failure, typically in the 

18 late teens or twenties. 

19 (3) Myotonic muscular dystrophy is the second 

20 most prominent form of muscular dystrophy and the 

21 type most commonly found in adults affecting an es22 timated 1 in 8,000 people. However, it can affect 

23 people of any age—from birth to old age. Described 

24 as the most variable disease known in medicine, it 

25 is multi-systemic and can cause not only muscle at26 rophy and myotonia, but also serious cardiac, resVerDate Aug 31 2005 04:58 Sep 28, 2008 Jkt 069200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H5265.RDS H5265

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1 piratory, endocrine, gastrointestinal, skeletal and 

2 central nervous system complications, as well as 

3 problems with the eyes, teeth and hair. As it passes 

4 from one generation to the next, it generally worsens 

5 with earlier onset. Congenital myotonic muscular 

6 dystrophy is the most severe form of myotonic mus7 cular dystrophy affecting infants and causing severe 

8 cognitive delays. It often causes sudden death; how9 ever, others can live for many years with this slowly 

10 degenerative disorder. 

11 (4) Facioscapulohumeral muscular dystrophy 

12 (referred to in this section as ‘‘FSHD’’) is the sec13 ond most prevalent adult muscular dystrophy and 

14 the third most prevalent muscular dystrophy of men, 

15 women and children. It is inherited genetically and 

16 has an estimated incidence of 1 in 20,000 persons. 

17 Many leading FSHD scientists note that the preva18 lence may be three times higher due to undiagnosed 

19 and misdiagnosed cases. FSHD, affecting between 

20 15,000 to 40,000 persons, causes a lifelong progres21 sive and severe loss of all skeletal muscles gradually 

22 bringing weakness and reduced mobility. It is geneti23 cally transmitted to children, can occur spontane24 ously, and may affect entire families. Persons with 

25 FSHD may also experience hearing loss, vision probVerDate Aug 31 2005 04:58 Sep 28, 2008 Jkt 069200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H5265.RDS H5265

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1 lems and respiratory insufficiency; some may become 

2 severely physically disabled and spend decades in a 

3 wheelchair and on a ventilator. FSHD is caused by 

4 a novel epigenetic phenomenon not found in other 

5 forms of muscular dystrophy and is caused by a con6 traction of repetitive DNA previously thought to be 

7 ‘‘junk DNA’’. The unique epigenetic structure of 

8 FSHD is unprecedented in other muscular dys9 trophies and genetic disorders and demands novel 

10 approaches and new research groups. Understanding 

11 this mechanism will have great benefit to other areas 

12 of biomedical research including cancer and other 

13 disease of epigenetic origin. 

14 (5) Congenital muscular dystrophies represent a 

15 group of distinct diseases, which begin at birth, with 

16 varying severity and involvement of both muscle 

17 strength and brain. These diseases often lead to pre18 mature infant death, or severely disabled young chil19 dren who require 24-hour care given their develop20 mental delay compounded by muscle weakness. 

21 Other children live to young adulthood and typically 

22 require the use of a wheelchair for mobility. 

23 (6) Forms of muscular dystrophy affecting chil24 dren and adults include Becker, congenital, distal, 

25 Duchenne, Emery-Dreifuss, facioscapulohumeral, 


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1 limb-girdle, myotonic, and oculopharyngeal muscular 

2 dystrophies. The limb-girdle muscular dystrophies 

3 are of 15 known different types. 

4 (7) Each of the muscular dystrophies, though 

5 distinct in progressivity and severity of symptoms, 

6 has a devastating impact on hundreds of thousands 

7 of children and adults throughout the United States 

8 and worldwide, as well as imposes severe physical 

9 and economic burdens on those affected. In many of 

10 the muscular dystrophies, there are associated med11 ical problems arising from pulmonary issues, res12 piratory insufficiency, cardiomyopathy, which in 

13 many cases is the cause of death for persons with 

14 muscular dystrophy. 

15 (8) In the 5 years since enactment of the Mus16 cular Dystrophy Community Assistance, Research 

17 and Education Amendments of 2001 (MD–CARE 

18 Act) and due directly to the momentum established 

19 by the MD–CARE Act, progress has been made in 

20 the battle against the Muscular Dystrophies. 

21 (9) Investments made by the Federal Govern22 ment as a result of the MD–CARE Act include the 

23 creation of the MD Coordinating Committee 

24 (MDCC), the development of the MDCC Action 

25 Plan, establishment of 6 Paul D. Wellstone MusVerDate Aug 31 2005 04:58 Sep 28, 2008 Jkt 069200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H5265.RDS H5265

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1 cular Dystrophy Cooperative Research Centers (co2 funded, in part, by a national non-profit health orga3 nization), development of the Muscular Dystrophy 

4 Surveillance, Tracking and Research Network (MD 

5 STARnet), and the launch of a comprehensive edu6 cation and outreach initiative. 

7 (10) In the past few years, the NIH program 

8 in translational research in muscular dystrophy has 

9 grown significantly and funded a number of large10 scale projects to further the development of thera11 pies for muscular dystrophy. As part of this pro12 gram, the National Institute of Neurological Dis13 orders and Stroke (NINDS) and the National Insti14 tute of Arthritis and Musculoskeletal and Skin Dis15 eases (NIAMS) of the National Institutes of Health 

16 (NIH) awarded a $15.4 million, five-year cooperative 

17 agreement to develop new small molecule drugs for 

18 the treatment of Duchenne muscular dystrophy 

19 (DMD) and potentially other forms of muscular dys20 trophy as well. The project is a unique research col21 laboration between private, public, and non-profit 

22 partners to build upon previous research and dis23 covery work originally initiated by non-profit part24 ners to identify new treatments for muscular dys25 trophy. Also through the translational program, 


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1 three other major cooperative agreements have been 

2 awarded for highly targeted therapy development 

3 projects in the muscular dystrophies. 

4 (11) Advancements in care have helped prolong 

5 life and quality of life for patients with muscular 

6 dystrophy. 

7 (12) There remains a shortage of qualified re8 searchers in the field of muscular dystrophy re9 search. Many family physicians and health care pro10 fessionals still lack the knowledge and resources to 

11 detect and properly diagnose muscular dystrophy as 

12 early as possible, thus delaying management of 

13 symptoms in cases that go undetected or 

14 misdiagnosed. 

15 (13) As new understandings of the genetic basis 

16 for disease and potential treatment has emerged, the 

17 public and health care communities are in urgent 

18 need of education and outreach to ensure competent, 

19 informed engagement in genetic testing and coun20 seling and appropriate patient characterization so 

21 that patients are able to participate in new avenues 

22 of research and clinical trials. 

23 (14) As basic research into the muscular dys24 trophies points the way to new therapeutic targets, 

25 there is an urgent need to support the clinical reVerDate Aug 31 2005 04:58 Sep 28, 2008 Jkt 069200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\H5265.RDS H5265

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1 search infrastructure necessary to bring these thera2 peutic leads to human trials; these infrastructure 

3 needs include validated endpoints, current natural 

4 history studies, biomarkers, clinical research net5 works, patient registries and databases. 

6 (15) In order to improve lives and develop effec7 tive treatments for individuals with muscular dys8 trophy, there must be improved communications and 

9 partnerships between patients, patient advocacy, re10 searchers, and clinical care providers. To that end, 

11 renewed effort to work together by all parties is a 

12 critical element for successful outcomes in the years 

13 to come. 

14 (16) Continued focus and investment are re15 quired to build on the current momentum, respond 

16 to public need, and ensure that research and other 

17 innovation is translated to therapeutic targets as 

18 quickly as possible. 

19 SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINA20 TION OF ACTIVITIES OF NIH WITH RESPECT 

21 TO RESEARCH ON MUSCULAR DYSTROPHY. 

22 (a) TECHNICAL CORRECTION.—Section 404E of the 

23 Public Health Service Act (42 U.S.C. 283g) is amended 

24 by striking subsection (f) (relating to reports to Congress) 

25 and redesignating subsection (g) as subsection (f). 


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1 (b) AMENDMENTS.—Section 404E of the Public 

2 Health Service Act (42 U.S.C. 283g) is amended— 

3 (1) in subsection (a)(1), by inserting ‘‘the Na4 tional Heart, Lung, and Blood Institute,’’ after ‘‘the 

5 Eunice Kennedy Shriver National Institute of Child 

6 Health and Human Development,’’; 

7 (2) in subsection (b)(1), by adding at the end 

8 of the following: ‘‘Such centers of excellence shall be 

9 known as the ‘Paul D. Wellstone Muscular Dys10 trophy Cooperative Research Centers’.’’; and 

11 (3) by adding at the end the following: 

12 ‘‘(g) CLINICAL RESEARCH.—The Coordinating Com13 mittee may evaluate the potential need to enhance the clin14 ical research infrastructure required to test emerging 

15 therapies for the various forms of muscular dystrophy by 

16 prioritizing the achievement of the goals related to this 

17 topic in the plan under subsection (e)(1).’’. 

18 SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF 

19 CDC WITH RESPECT TO EPIDEMIOLOGICAL 

20 RESEARCH ON MUSCULAR DYSTROPHY. 

21 Section 317Q of the Public Health Service Act (42 

22 U.S.C. 247b–18) is amended— 

23 (1) by redesignating subsection (d) as sub24 section (f); and 


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1 (2) by inserting after subsection (c) the fol2 lowing: 

3 ‘‘(d) DATA.—In carrying out this section, the Sec4 retary shall ensure that any data on patients that is col5 lected as part of the Muscular Dystrophy STARnet (under 

6 a grant under this section) is regularly updated to reflect 

7 changes in patient condition over time. 

8 ‘‘(e) REPORTS AND STUDY.— 

9 ‘‘(1) ANNUAL REPORT.—Not later than 18 

10 months after the date of the enactment of the Paul 

11 D. Wellstone Muscular Dystrophy Community As12 sistance, Research, and Education Amendments of 

13 2008, and annually thereafter, the Director of the 

14 Centers for Disease Control and Prevention shall 

15 submit to the appropriate committees of the Con16 gress a report— 

17 ‘‘(A) concerning the activities carried out 

18 by MD STARnet site funded under this section 

19 during the year for which the report is pre20 pared; 

21 ‘‘(B) containing the data collected and 

22 findings derived from the MD STARnet sites 

23 each fiscal year (as funded under a grant under 

24 this section during fiscal years 2008 through 

25 2012); and 


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1 ‘‘(C) that every 2 years outlines prospec2 tive data collection objectives and strategies. 

3 ‘‘(2) TRACKING HEALTH OUTCOMES.—The Di4 rector of the Centers for Disease Control and Pre5 vention shall provide health outcome data on the 

6 health and survival of people with muscular dys7 trophy.’’. 

8 SEC. 5. INFORMATION AND EDUCATION. 

9 Section 5 of the Muscular Dystrophy Community As10 sistance, Research and Education Amendments of 2001 

11 (42 U.S.C. 247b–19) is amended— 

12 (1) by redesignating subsection (c) as sub13 section (d); and 

14 (2) by inserting after subsection (b) the fol15 lowing: 

16 ‘‘(c) REQUIREMENTS OF CDC.—In carrying out this 

17 section, the Director of the Centers for Disease Control 

18 and Prevention shall— 

19 ‘‘(1) partner with leaders in the muscular dys20 trophy patient community; and 

21 ‘‘(2) widely disseminate the Duchenne-Becker 

22 muscular dystrophy care considerations as broadly 

23 as possible, including through partnership opportuni24 ties with the muscular dystrophy patient commu25 nity.’’. 


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1 SEC. 6. STANDARDS OF CARE. 

2 Part A of title IX of the Public Health Service Act 

3 (42 U.S.C. 299 et seq.) is amended by adding at the end 

4 the following: 

5 ‘‘SEC. 904. STANDARDS OF CARE RELATING TO MUSCULAR 

6 DYSTROPHY. 

7 ‘‘The Director— 

8 ‘‘(1) shall evaluate the available scientific evi9 dence for the appropriate medical or patient organi10 zations for purposes of the development and 

11 issuance of an initial set of care considerations for 

12 Duchenne-Becker muscular dystrophy and provide 

13 periodic review and updates where appropriate; and 

14 ‘‘(2) may replicate the same methodology used 

15 to develop the Duchenne-Becker muscular dystrophy 

16 care considerations developed under paragraph (1) 

17 as a model for other muscular dystrophies.’’. 

Passed the House of Representatives September 24, 

2008. 

Attest: LORRAINE C. MILLER, 

Clerk. 


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