II Calendar No. 478 113th CONGRESS 2d Session S. 315 IN THE SENATE OF THE UNITED STATES February 13, 2013 Ms. Klobuchar (for herself, Mr. Wicker , Mr. Sanders , Ms. Collins , Mr. Menendez , Mr. Isakson , Ms. Mikulski , Mr. Leahy , Mr. Lautenberg , Mr. Nelson , Mr. Schumer , Mr. Cochran , Mrs. Boxer , Mr. King , Mr. Bennet , Ms. Stabenow , Mr. Blunt , Mrs. Shaheen , Mr. Markey , Mrs. Gillibrand , Mr. Brown , Mr. Tester , Mr. Graham , Mr. Coons , Ms. Warren , Ms. Cantwell , Mr. Pryor , Mr. Booker , Mr. Franken , and Mr. Harkin ) introduced the following bill; which was read twice and referred to the Committee on Health, Education, Labor, and Pensions July 23, 2014 Reported by Mr. Harkin , with an amendment and an amendment to the title Strike out all after the enacting clause and insert the part printed in italic A BILL To reauthorize and extend the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2008.

1. Short title This Act may be cited as the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education (MD–CARE) Amendments of 2013 . 2. Initiative through the Director of the National Institutes of Health Section 404E of the Public Health Service Act ( 42 U.S.C. 283g ) is amended— (1) in subsection (a)(1)— (A) by striking Muscoskeletal and inserting Musculoskeletal ; and (B) by inserting Becker, myotonic, facioscapulohumeral muscular dystrophy (referred to in this section as FSHD ), Congenital muscular dystrophy, Limb-girdle muscular dystrophy, after Duchenne, ; (2) in subsection (b)— (A) in paragraph (2), by inserting cardiac and pulmonary function, after imaging, genetics, ; and (B) in paragraph (3), by inserting and sharing of data after regular communication ; (3) in subsection (d)— (A) in paragraph (2)— (i) in the matter preceding subparagraph (A), by striking 15 and inserting 18 ; and (ii) in subparagraph (A), by striking children with muscular dystrophy, such as the Department of Education and inserting children and adults with muscular dystrophy, such as the Department of Education, the Social Security Administration, the United States Administration for Community Living ; and (B) in paragraph (4)(B), by inserting , and shall meet no less than two times per calendar year before the period; (4) in subsection (e)— (A) in paragraph (1)— (i) in the matter preceding subparagraph (A), by striking through the national research institutes and inserting through the represented Federal agencies ; and (ii) in subparagraph (A), by striking and rehabilitative issues, including studies of the impact of such diseases in rural and underserved communities and inserting public resources, and rehabilitative issues, including studies of the impact of such diseases in rural and underserved communities, health economic studies to demonstrate the cost-effectiveness of providing independent living resources and support to patients with various forms of muscular dystrophy, and studies to determine optimal clinical care interventions for adults with various forms of muscular dystrophy ; and (B) in paragraph (2), by adding at the end the following: (F) The development of clinical interventions to improve the health of adults with various forms of muscular dystrophy. ; and (5) in subsection (g), by striking 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) and inserting and shall, not later than 6 months after the date of enactment of the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education (MD–CARE) Amendments of 2013 , in coordination with appropriate Federal agencies, including relevant offices within the Food and Drug Administration and supported by the National Institutes of Health and Department of Defense, develop a plan to expedite the evaluation and approval of emerging therapies and personalized medicines that have the potential to decrease fatal disease progression across the various forms of muscular dystrophy . 3. Surveillance and research regarding muscular dystrophy Section 317Q of the Public Health Service Act ( 42 U.S.C. 247b–18 ) is amended— (1) in the second sentence of subsection (b), by inserting before the period the following: and ensure that the program captures data from different racial and ethnic populations, and that such data are made publicly available to investigators conducting public or private research on muscular dystrophy ; and (2) in subsection (c), by adding at the end the following: The Secretary shall also foster ongoing engagement and collaboration between the surveillance program and centers of excellence. . 4. Information and education Section 5(c) of the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 ( 42 U.S.C. 247b–19(c) ) is amended by adding at the end the following: (4) Update and widely disseminate existing Duchenne-Becker muscular dystrophy care considerations for pediatric patients, develop and widely disseminate Duchenne-Becker muscular dystrophy care considerations for adult patients, and develop and widely disseminate acute care considerations for all muscular dystrophy populations. The care considerations should build upon existing efforts currently underway for congenital muscular dystrophy, fascioscapulohumeral muscular dystrophy, limb-girdle muscular dystrophy, and myotonic muscular dystrophy, and incorporate strategies specifically responding to the findings of the national transitions survey of minority, young adult and adult communities of muscular dystrophy patients. .

1. Short title This Act may be cited as the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2014 . 2. Initiative through the Director of the National Institutes of Health Section 404E of the Public Health Service Act ( 42 U.S.C. 283g ) is amended— (1) in subsection (a)(1)— (A) by striking Muscoskeletal and inserting Musculoskeletal ; and (B) by inserting Becker, congenital muscular dystrophy, limb-girdle muscular dystrophy, after Duchenne, ; (2) in subsection (b)— (A) in paragraph (2)— (i) by striking genetics, at the second place it appears; and (ii) by inserting cardiac and pulmonary function, and after imaging, ; and (B) in paragraph (3), by inserting and sharing of data after regular communication ; (3) in subsection (d)— (A) in paragraph (2)— (i) in the matter preceding subparagraph (A), by striking 15 and inserting 18 ; and (ii) in subparagraph (A)— (I) by striking and the Food and Drug Administration and inserting , the Food and Drug Administration, and the Administration for Community Living ; (II) by inserting and adults after children ; and (III) by striking such as the Department of Education and inserting including the Department of Education and the Social Security Administration ; and (B) in paragraph (4)(B), by inserting , but shall meet no fewer than two times per calendar year before the period; and (4) in subsection (e)— (A) in paragraph (1)— (i) in the matter preceding subparagraph (A), by striking through the national research institutes and inserting through the agencies represented on the Coordinating Committee pursuant to subsection (d)(2)(A) ; and (ii) in subparagraph (A)— (I) by inserting public services, before and rehabilitative issues ; and (II) by inserting , studies to demonstrate the cost-effectiveness of providing independent living resources and support to patients with various forms of muscular dystrophy, and studies to determine optimal clinical care interventions for adults with various forms of muscular dystrophy after including studies of the impact of such diseases in rural and underserved communities ; and (B) in paragraph (2)(D), by inserting after including new biological agents the following: and new clinical interventions to improve the health of those with muscular dystrophy . 3. Surveillance and research regarding muscular dystrophy The second sentence of section 317Q(b) of the Public Health Service Act ( 42 U.S.C. 247b–18(b) ) is amended by inserting before the period the following: and, to the extent possible, ensure that data be representative of all affected populations and shared in a timely manner . 4. Information and education Section 5(c) of the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 ( 42 U.S.C. 247b–19(c) ) is amended— (1) in paragraph (2)— (A) by inserting for pediatric and adult patients, including acute care considerations, after issuance of care considerations ; (B) by inserting various before other forms of muscular dystrophy ; and (C) by striking and at the end; (2) by redesignating paragraph (3) as paragraph (4); (3) by inserting after paragraph (2) the following: (3) in developing and updating care considerations under paragraph (2), incorporate strategies specifically responding to the findings of the national transitions survey of minority, young adult, and adult communities of muscular dystrophy patients; and ; and (4) in paragraph (4), as redesignated, by inserting various before other forms of muscular dystrophy .

Amend the title so as to read: A bill to amend the Public Health Service Act relating to Federal research on muscular dystrophy, and for other purposes. .

July 23, 2014 Reported with an amendment and an amendment to the title