[Congressional Record Volume 154, Number 151 (Tuesday, September 23, 2008)]
[House]
[Pages H8676-H8678]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




 PAUL D. WELLSTONE MUSCULAR DYSTROPHY COMMUNITY ASSISTANCE, RESEARCH, 
                    AND EDUCATION AMENDMENTS OF 2008

  Mr. PALLONE. Mr. Speaker, I move to suspend the rules and pass the 
bill (H.R. 5265) 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, as amended.
  The Clerk read the title of the bill.
  The text of the bill is as follows:

                               H.R. 5265

       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 ``Paul D. Wellstone Muscular 
     Dystrophy Community Assistance, Research, and Education 
     Amendments of 2008''.

     SEC. 2. FINDINGS.

       The Congress finds as follows:
       (1) The muscular dystrophies are devastating diseases that 
     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.
       (2) DMD is the most common lethal genetic disorder of 
     childhood worldwide, affecting approximately 1 in every 3,500 
     boys born each year around the globe. It is characterized by 
     a rapidly progressive muscle weakness that almost always 
     results in death from respiratory or cardiac failure, 
     typically in the late teens or twenties.
       (3) Myotonic muscular dystrophy is the second most 
     prominent form of muscular dystrophy and the type most 
     commonly found in adults affecting an estimated 1 in 8,000 
     people. However, it can affect people of any age--from birth 
     to old age. Described as the most variable disease known in 
     medicine, it is multi-systemic and can cause not only muscle 
     atrophy and myotonia, but also serious cardiac, respiratory, 
     endocrine, gastrointestinal, skeletal and central nervous 
     system complications, as well as problems with the eyes, 
     teeth and hair. As it passes from one generation to the next, 
     it generally worsens with earlier onset. Congenital myotonic 
     muscular dystrophy is the most severe form of myotonic 
     muscular dystrophy affecting infants and causing severe 
     cognitive delays. It often causes sudden death; however, 
     others can live for many years with this slowly degenerative 
     disorder.
       (4) Facioscapulohumeral muscular dystrophy (referred to in 
     this section as ``FSHD'') is the second most prevalent adult 
     muscular dystrophy and the third most prevalent muscular 
     dystrophy of men, women and children. It is inherited 
     genetically and has an estimated incidence of 1 in 20,000 
     persons. Many leading FSHD scientists note

[[Page H8677]]

     that the prevalence may be three times higher due to 
     undiagnosed and misdiagnosed cases. FSHD, affecting between 
     15,000 to 40,000 persons, causes a lifelong progressive and 
     severe loss of all skeletal muscles gradually bringing 
     weakness and reduced mobility. It is genetically transmitted 
     to children, can occur spontaneously, and may affect entire 
     families. Persons with FSHD may also experience hearing loss, 
     vision problems and respiratory insufficiency; some may 
     become severely physically disabled and spend decades in a 
     wheelchair and on a ventilator. FSHD is caused by a novel 
     epigenetic phenomenon not found in other forms of muscular 
     dystrophy and is caused by a contraction of repetitive DNA 
     previously thought to be ``junk DNA''. The unique epigenetic 
     structure of FSHD is unprecedented in other muscular 
     dystrophies and genetic disorders and demands novel 
     approaches and new research groups. Understanding this 
     mechanism will have great benefit to other areas of 
     biomedical research including cancer and other disease of 
     epigenetic origin.
       (5) Congenital muscular dystrophies represent a group of 
     distinct diseases, which begin at birth, with varying 
     severity and involvement of both muscle strength and brain. 
     These diseases often lead to premature infant death, or 
     severely disabled young children who require 24-hour care 
     given their developmental delay compounded by muscle 
     weakness. Other children live to young adulthood and 
     typically require the use of a wheelchair for mobility.
       (6) Forms of muscular dystrophy affecting children and 
     adults include Becker, congenital, distal, Duchenne, Emery-
     Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and 
     oculopharyngeal muscular dystrophies. The limb-girdle 
     muscular dystrophies are of 15 known different types.
       (7) Each of the muscular dystrophies, though distinct in 
     progressivity and severity of symptoms, has a devastating 
     impact on hundreds of thousands of children and adults 
     throughout the United States and worldwide, as well as 
     imposes severe physical and economic burdens on those 
     affected. In many of the muscular dystrophies, there are 
     associated medical problems arising from pulmonary issues, 
     respiratory insufficiency, cardiomyopathy, which in many 
     cases is the cause of death for persons with muscular 
     dystrophy.
       (8) In the 5 years since enactment of the Muscular 
     Dystrophy Community Assistance, Research and Education 
     Amendments of 2001 (MD-CARE Act) and due directly to the 
     momentum established by the MD-CARE Act, progress has been 
     made in the battle against the Muscular Dystrophies.
       (9) Investments made by the Federal Government as a result 
     of the MD-CARE Act include the creation of the MD 
     Coordinating Committee (MDCC), the development of the MDCC 
     Action Plan, establishment of 6 Paul D. Wellstone Muscular 
     Dystrophy Cooperative Research Centers (co-funded, in part, 
     by a national non-profit health organization), development of 
     the Muscular Dystrophy Surveillance, Tracking and Research 
     Network (MD STARnet), and the launch of a comprehensive 
     education and outreach initiative.
       (10) In the past few years, the NIH program in 
     translational research in muscular dystrophy has grown 
     significantly and funded a number of large-scale projects to 
     further the development of therapies for muscular dystrophy. 
     As part of this program, the National Institute of 
     Neurological Disorders and Stroke (NINDS) and the National 
     Institute of Arthritis and Musculoskeletal and Skin Diseases 
     (NIAMS) of the National Institutes of Health (NIH) awarded a 
     $15.4 million, five-year cooperative agreement to develop new 
     small molecule drugs for the treatment of Duchenne muscular 
     dystrophy (DMD) and potentially other forms of muscular 
     dystrophy as well. The project is a unique research 
     collaboration between private, public, and non-profit 
     partners to build upon previous research and discovery work 
     originally initiated by non-profit partners to identify new 
     treatments for muscular dystrophy. Also through the 
     translational program, three other major cooperative 
     agreements have been awarded for highly targeted therapy 
     development projects in the muscular dystrophies.
       (11) Advancements in care have helped prolong life and 
     quality of life for patients with muscular dystrophy.
       (12) There remains a shortage of qualified researchers in 
     the field of muscular dystrophy research. Many family 
     physicians and health care professionals still lack the 
     knowledge and resources to detect and properly diagnose 
     muscular dystrophy as early as possible, thus delaying 
     management of symptoms in cases that go undetected or 
     misdiagnosed.
       (13) As new understandings of the genetic basis for disease 
     and potential treatment has emerged, the public and health 
     care communities are in urgent need of education and outreach 
     to ensure competent, informed engagement in genetic testing 
     and counseling and appropriate patient characterization so 
     that patients are able to participate in new avenues of 
     research and clinical trials.
       (14) As basic research into the muscular dystrophies points 
     the way to new therapeutic targets, there is an urgent need 
     to support the clinical research infrastructure necessary to 
     bring these therapeutic leads to human trials; these 
     infrastructure needs include validated endpoints, current 
     natural history studies, biomarkers, clinical research 
     networks, patient registries and databases.
       (15) In order to improve lives and develop effective 
     treatments for individuals with muscular dystrophy, there 
     must be improved communications and partnerships between 
     patients, patient advocacy, researchers, and clinical care 
     providers. To that end, renewed effort to work together by 
     all parties is a critical element for successful outcomes in 
     the years to come.
       (16) Continued focus and investment are required to build 
     on the current momentum, respond to public need, and ensure 
     that research and other innovation is translated to 
     therapeutic targets as quickly as possible.

     SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINATION OF 
                   ACTIVITIES OF NIH WITH RESPECT TO RESEARCH ON 
                   MUSCULAR DYSTROPHY.

       (a) Technical Correction.--Section 404E of the Public 
     Health Service Act (42 U.S.C. 283g) is amended by striking 
     subsection (f) (relating to reports to Congress) and 
     redesignating subsection (g) as subsection (f).
       (b) Amendments.--Section 404E of the Public Health Service 
     Act (42 U.S.C. 283g) is amended--
       (1) in subsection (a)(1), by inserting ``the National 
     Heart, Lung, and Blood Institute,'' after ``the Eunice 
     Kennedy Shriver National Institute of Child Health and Human 
     Development,'';
       (2) in subsection (b)(1), by adding at the end of the 
     following: ``Such centers of excellence shall be known as the 
     `Paul D. Wellstone Muscular Dystrophy Cooperative Research 
     Centers'.''; and
       (3) by adding at the end the following:
       ``(g) Clinical Research.--The Coordinating Committee may 
     evaluate the potential need to enhance the clinical research 
     infrastructure required to test emerging therapies for the 
     various forms of muscular dystrophy by prioritizing the 
     achievement of the goals related to this topic in the plan 
     under subsection (e)(1).''.

     SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF CDC WITH 
                   RESPECT TO EPIDEMIOLOGICAL RESEARCH ON MUSCULAR 
                   DYSTROPHY.

       Section 317Q of the Public Health Service Act (42 U.S.C. 
     247b-18) is amended--
       (1) by redesignating subsection (d) as subsection (f); and
       (2) by inserting after subsection (c) the following:
       ``(d) Data.--In carrying out this section, the Secretary 
     shall ensure that any data on patients that is collected as 
     part of the Muscular Dystrophy STARnet (under a grant under 
     this section) is regularly updated to reflect changes in 
     patient condition over time.
       ``(e) Reports and Study.--
       ``(1) Annual report.--Not later than 18 months after the 
     date of the enactment of the Paul D. Wellstone Muscular 
     Dystrophy Community Assistance, Research, and Education 
     Amendments of 2008, and annually thereafter, the Director of 
     the Centers for Disease Control and Prevention shall submit 
     to the appropriate committees of the Congress a report--
       ``(A) concerning the activities carried out by MD STARnet 
     site funded under this section during the year for which the 
     report is prepared;
       ``(B) containing the data collected and findings derived 
     from the MD STARnet sites each fiscal year (as funded under a 
     grant under this section during fiscal years 2008 through 
     2012); and
       ``(C) that every 2 years outlines prospective data 
     collection objectives and strategies.
       ``(2) Tracking health outcomes.--The Director of the 
     Centers for Disease Control and Prevention shall provide 
     health outcome data on the health and survival of people with 
     muscular dystrophy.''.

     SEC. 5. INFORMATION AND EDUCATION.

       Section 5 of the Muscular Dystrophy Community Assistance, 
     Research and Education Amendments of 2001 (42 U.S.C. 247b-19) 
     is amended--
       (1) by redesignating subsection (c) as subsection (d); and
       (2) by inserting after subsection (b) the following:
       ``(c) Requirements of CDC.--In carrying out this section, 
     the Director of the Centers for Disease Control and 
     Prevention shall--
       ``(1) partner with leaders in the muscular dystrophy 
     patient community; and
       ``(2) widely disseminate the Duchenne-Becker muscular 
     dystrophy care considerations as broadly as possible, 
     including through partnership opportunities with the muscular 
     dystrophy patient community.''.

     SEC. 6. STANDARDS OF CARE.

       Part A of title IX of the Public Health Service Act (42 
     U.S.C. 299 et seq.) is amended by adding at the end the 
     following:

     ``SEC. 904. STANDARDS OF CARE RELATING TO MUSCULAR DYSTROPHY.

       ``The Director--
       ``(1) shall evaluate the available scientific evidence for 
     the appropriate medical or patient organizations for purposes 
     of the development and issuance of an initial set of care 
     considerations for Duchenne-Becker muscular dystrophy and 
     provide periodic review and updates where appropriate; and
       ``(2) may replicate the same methodology used to develop 
     the Duchenne-Becker muscular dystrophy care considerations 
     developed under paragraph (1) as a model for other muscular 
     dystrophies.''.

  The SPEAKER pro tempore. Pursuant to the rule, the gentleman from

[[Page H8678]]

New Jersey (Mr. Pallone) and the gentleman from Texas (Mr. Burgess) 
each will control 20 minutes.
  The Chair recognizes the gentleman from New Jersey.


                             General Leave

  Mr. PALLONE. Mr. Speaker, I ask unanimous consent that all Members 
may have 5 legislative days to revise and extend their remarks and 
include extraneous material on the bill under consideration.
  The SPEAKER pro tempore. Is there objection to the request of the 
gentleman from New Jersey?
  There was no objection.
  Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
  Mr. Speaker, I rise in support of H.R. 5265, the Paul Wellstone 
Muscular Dystrophy Community Assistance, Research, and Education 
Amendments of 2008, a bill to reauthorize programs at the National 
Institutes of Health and the Centers for Disease Control and Prevention 
for research on various forms of muscular dystrophy.
  Duchenne-Becker muscular dystrophy, DBMD, is a combined spectrum of a 
genetic disorder. DBMD is usually diagnosed when the child is 3 to 6 
years of age. Early signs include delays in walking and frequent 
falling. As the child grows older, muscle deterioration continues to 
progress until, finally, the disease reaches a fatal conclusion in the 
teen years.
  Enacting H.R. 5265 would make a number of improvements to current 
programs at the NIH and CDC. It would allow the interagency 
coordinating committee for muscular dystrophy to give special 
consideration to enhancing the clinical research infrastructure 
required to test emerging therapies for the various forms of muscular 
dystrophy, require the director of the CDC to report on muscular 
dystrophy surveillance, tracking, and research network data collection 
and provide for respective health outcome data on the health and 
survival of people with muscular dystrophy and require the director of 
the Agency for Health Care Research and Quality to evaluate the 
available scientific evidence to develop and issue an initial set of 
care considerations for DBMD and provide ongoing review and updates. 
All of the above coordinated research and tracking efforts will 
continue to lead us down a path towards one day finding a cure for this 
tragic condition.
  I want to thank my colleague on the Commerce Committee, Congressman 
Engel of New York, for his leadership on this legislation. He has been 
working on this bill for a long time, and I appreciate his efforts to 
craft a strong bipartisan product.
  I fully support H.R. 5265 and urge my colleagues to join me in its 
adoption.
  Mr. Speaker, I reserve the balance of my time.
  Mr. BURGESS. Mr. Speaker, I yield myself such time as I may consume, 
and I rise in support of H.R. 5265, the Paul D. Wellstone Muscular 
Dystrophy Community Assistance, Research, and Education Amendments of 
2008. I want to commend Congressman Engel for bringing this bill, and I 
was also proud to be the lead minority cosponsor on this bill.
  H.R. 5265 reauthorizes the existing Centers for Disease Control 
efforts towards muscular dystrophy. The muscular dystrophies are a 
group of more than 30 genetic diseases characterized by progressive 
weakness and degeneration of the skeletal muscles that control 
voluntary movement. Muscular dystrophy funding is used for surveillance 
and family needs and assessment activities.
  I am supportive of the bill's efforts to reauthorize and improve the 
existing muscular dystrophy registry at the Centers for Disease 
Control, and I urge Members to support the legislation.
  Mr. Speaker, I yield back the balance my time.

                              {time}  2030

  Mr. PALLONE. Mr. Speaker, I have no further requests for time, and I 
urge adoption of this bill, the Paul Wellstone Muscular Dystrophy 
Community Assistance, Research, and Education Amendments of 2008.
  Mr. ENGEL. Mr. Speaker, I am so proud that today we will move to pass 
H.R. 5265, the Paul D. Wellstone Muscular Dystrophy Community 
Assistance, Research, and Education Amendments of 2008. Today we have 
over 120 bipartisan cosponsors, as well as the support of the medical 
community.
  Muscular dystrophy is a genetic disease which results in progressive 
degeneration of skeletal muscles and other organs, notably the heart. 
There are nine muscular dystrophies affecting over 300,000 individuals 
in the United States. The most lethal is Duchenne muscular dystrophy, 
which affects 1 in every 3,500 boys. There is no cure.
  Prior to 2001, there were few resources directed toward research and 
development of therapies and care models for those afflicted with 
muscular dystrophy. To address this issue, the Muscular Dystrophy 
Community Assistance, Research and Education Amendments of 2001 was 
introduced. Congress overwhelmingly supported the legislation. 
Unfortunately, the authorization for this work expired in 2006.
  The 2001 law specified a number of provisions for expanding and 
intensifying research on muscular dystrophy. These efforts included the 
establishment of six scientific centers of excellence, the creation of 
a Muscular Dystrophy Coordinating Committee (MDCC) to develop plans for 
supporting research and education on muscular dystrophy, and an 
expansion by the Centers for Disease Control and Prevention (CDC) into 
epidemiological activities regarding muscular dystrophy.
  The reauthorization of the Paul D. Wellstone MD-CARE Amendments 
officially names the Centers of Excellence the Paul D. Wellstone 
Muscular Dystrophy Cooperative Research Centers. In addition, it 
ensures that data collection at CDC is updated regularly with a 
requirement for regular reports to Congress. The bill also requires the 
Agency for Healthcare Research and Quality to work with appropriate 
medical or patient organizations to finalize an initial set of care 
considerations and for CDC to disseminate that information to targeted 
audiences.
  Once again, thank you Mr. Speaker for your commitment to muscular 
dystrophy.
  I know this bill will have a profound effect on so many families in 
America upon enactment.
  Mr. PALLONE. I yield back the balance of my time.
  The SPEAKER pro tempore. The question is on the motion offered by the 
gentleman from New Jersey (Mr. Pallone) that the House suspend the 
rules and pass the bill, H.R. 5265, as amended.
  The question was taken.
  The SPEAKER pro tempore. In the opinion of the Chair, two-thirds 
being in the affirmative, the ayes have it.
  Mr. BURGESS. Mr. Speaker, I object to the vote on the ground that a 
quorum is not present and make the point of order that a quorum is not 
present.
  The SPEAKER pro tempore. Pursuant to clause 8 of rule XX and the 
Chair's prior announcement, further proceedings on this motion will be 
postponed.
  The point of no quorum is considered withdrawn.

                          ____________________