{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,13]],"date-time":"2026-02-13T14:49:39Z","timestamp":1770994179626,"version":"3.50.1"},"reference-count":20,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2010,2,6]],"date-time":"2010-02-06T00:00:00Z","timestamp":1265414400000},"content-version":"vor","delay-in-days":5,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2010,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Pulmonary function is impaired in untreated mucopolysaccharidosis type VI (MPS VI). Pulmonary function was studied in patients during long\u2010term enzyme replacement therapy (ERT) with recombinant human arylsulfatase B (rhASB; rh<jats:italic>N<\/jats:italic>\u2010acetylgalactosamine 4\u2010sulfatase). Pulmonary function tests prior to and for up to 240 weeks of weekly infusions of rhASB at 1 mg\/kg were completed in 56 patients during Phase 1\/2, Phase 2, Phase 3 and Phase 3 Extension trials of rhASB and the Survey Study. Forced vital capacity (FVC), forced expiratory volume in 1 s (FEV1) and, in a subset of patients, maximum voluntary ventilation (MVV), were analyzed as absolute volume in liters. FEV1 and FVC showed little change from baseline during the first 24 weeks of ERT, but after 96 weeks, these parameters increased over baseline by 11% and 17%, respectively. This positive trend compared with baseline continued beyond 96 weeks of treatment. Improvements from baseline in pulmonary function occurred along with gains in height in the younger group (5.5% change) and in the older patient group (2.4% change) at 96 weeks. Changes in MVV occurred earlier within 24 weeks of treatment to approximately 15% over baseline. Model results based on data from all trials showed significant improvements in the rate of change in pulmonary function during 96 weeks on ERT, whereas little or no improvement was observed for the same time period prior to ERT. Thus, analysis of mean percent change data and longitudinal modeling both indicate that long\u2010term ERT resulted in improvement in pulmonary function in MPS VI patients.<\/jats:p>","DOI":"10.1007\/s10545-009-9007-8","type":"journal-article","created":{"date-parts":[[2010,2,5]],"date-time":"2010-02-05T12:02:23Z","timestamp":1265371343000},"page":"51-60","source":"Crossref","is-referenced-by-count":78,"title":["Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long\u2010term pulmonary function in patients treated with recombinant human <i>N<\/i>\u2010acetylgalactosamine 4\u2010sulfatase"],"prefix":"10.1002","volume":"33","author":[{"given":"Paul","family":"Harmatz","sequence":"first","affiliation":[{"name":"Children\u2032s Hospital &amp; Research Center Oakland 747 52nd Street Oakland CA 94609 USA"}]},{"given":"Zi\u2010Fan","family":"Yu","sequence":"additional","affiliation":[{"name":"Statistics Collaborative, Inc Washington DC USA"}]},{"given":"Roberto","family":"Giugliani","sequence":"additional","affiliation":[{"name":"Medical Genetics Service\/HCPA and Department of Genetics\/UFRGS Porto Alegre Brazil"}]},{"given":"Ida Vanessa D.","family":"Schwartz","sequence":"additional","affiliation":[{"name":"Medical Genetics Service\/HCPA and Department of Genetics\/UFRGS Porto Alegre Brazil"}]},{"given":"Nathalie","family":"Guffon","sequence":"additional","affiliation":[{"name":"H\u00f4pital Femme M\u00e8re Enfant Service Maladies M\u00e9taboliques Bron France"}]},{"given":"Elisa Le\u00e3o","family":"Teles","sequence":"additional","affiliation":[{"name":"Departamento Pediatria, Hospital de Sao Jo\u00e3o Unidade de Doen\u00e7as Metab\u00f3licas Porto Portugal"}]},{"given":"M. Clara S\u00e1","family":"Miranda","sequence":"additional","affiliation":[{"name":"Instituto de Biologia Molecular e Celular Unidade de Biologia do Lisossoma e Peroxisoma Porto Portugal"}]},{"given":"J. Edmond","family":"Wraith","sequence":"additional","affiliation":[{"name":"Royal Manchester Children\u2032s Hosp Manchester UK"}]},{"given":"Michael","family":"Beck","sequence":"additional","affiliation":[{"name":"Children\u2032s Hosp University of Mainz Mainz Germany"}]},{"given":"Laila","family":"Arash","sequence":"additional","affiliation":[{"name":"Children\u2032s Hosp University of Mainz Mainz Germany"}]},{"given":"Maurizio","family":"Scarpa","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Padova Padova Italy"}]},{"given":"David","family":"Ketteridge","sequence":"additional","affiliation":[{"name":"SA Pathology at Women\u2032s and Children\u2032s Hospital North Adelaide SA Australia"}]},{"given":"John J.","family":"Hopwood","sequence":"additional","affiliation":[{"name":"Department of Genetic Medicine Women\u2032s and Children\u2032s Hospital Adelaide North Adelaide SA Australia"}]},{"given":"Barbara","family":"Plecko","sequence":"additional","affiliation":[{"name":"Univ. Klinik f\u00fcr Kinder und Jugendheilkunde Graz Austria"}]},{"given":"Robert","family":"Steiner","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Molecular and Medical Genetics Oregon Health &amp; Science University Portland OR USA"}]},{"given":"Chester B","family":"Whitley","sequence":"additional","affiliation":[{"name":"University of Minnesota Medical School Minneapolis MN USA"}]},{"given":"Paige","family":"Kaplan","sequence":"additional","affiliation":[{"name":"Children\u2032s Hospital of Philadelphia Philadelphia PA USA"}]},{"given":"Stuart J.","family":"Swiedler","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc Novato CA USA"}]},{"given":"Karen","family":"Hardy","sequence":"additional","affiliation":[{"name":"Children\u2032s Hospital &amp; Research Center Oakland 747 52nd Street Oakland CA 94609 USA"}]},{"given":"Kenneth I.","family":"Berger","sequence":"additional","affiliation":[{"name":"Departments of Medicine, Physiology and Neuroscience New York University School of Medicine New York NY USA"}]},{"given":"Celeste","family":"Decker","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc Novato CA USA"}]}],"member":"311","published-online":{"date-parts":[[2010,2,6]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1164\/ajrccm\/144.5.1202"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1164\/ajrccm.152.3.7663792"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1136\/thx.38.8.616"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2007-3847"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.0954-6820.1964.tb00940.x"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.1950200405"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2006-2184"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.03.018"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2004-1023"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2005.12.014"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2008.04.001"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1183\/09031936.05.00035205"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1136\/thx.48.8.794"},{"issue":"202","key":"e_1_2_9_15_1","first-page":"205","article-title":"Respiratory muscle function and ventilatory control. I in patients with motor neurone disease. II in patients with myotonic dystrophy","volume":"51","author":"Serisier DE","year":"1982","journal-title":"Q J Med"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ijporl.2005.01.017"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.10298"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1378\/chest.93.2.364"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1378\/chest.98.1.145"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.30579"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.01.046"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/s10545-009-9007-8","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1007\/s10545-009-9007-8","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/s10545-009-9007-8","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T14:23:31Z","timestamp":1694787811000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1007\/s10545-009-9007-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2010,2]]},"references-count":20,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2010,2]]}},"alternative-id":["10.1007\/s10545-009-9007-8"],"URL":"https:\/\/doi.org\/10.1007\/s10545-009-9007-8","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2010,2]]}}}