```

[Congressional Bills 110th Congress]
[From the U.S. Government Publishing Office]
[H.R. 5265 Received in Senate (RDS)]

  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.

    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 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.''.

            Passed the House of Representatives September 24, 2008.

            Attest:

                                            LORRAINE C. MILLER,

                                                                 Clerk.
```