{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,18]],"date-time":"2026-09-18T10:48:56Z","timestamp":1789728536423,"version":"4.0.1"},"reference-count":28,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2016,8,23]],"date-time":"2016-08-23T00:00:00Z","timestamp":1471910400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2016,8,23]],"date-time":"2016-08-23T00:00:00Z","timestamp":1471910400000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100004229","name":"Fondazione Pierfranco e Luisa Mariani","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004229","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["UL1 TR000004"],"award-info":[{"award-number":["UL1 TR000004"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008484","name":"BioMarin Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008484","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2016,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To present long\u2010term respiratory function outcomes from an open\u2010label, multi\u2010center, phase 3 extension study (MOR\u2010005) of elosulfase alfa enzyme replacement therapy (ERT) in patients with Morquio A syndrome.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>In part 1 of MOR\u2010005, patients initially randomized to ERT in the 24\u2010week pivotal study (MOR\u2010004) remained on their regimen (2.0 mg\/kg\/week or every other week); placebo patients were re\u2010randomized to one of the two regimens. During part 2, all patients received elosulfase alfa 2.0 mg\/kg\/week. Respiratory function was one of the efficacy endpoints evaluated in MOR\u2010005. Change from MOR\u2010004 baseline to 120 weeks of treatment for the combined population was determined and compared with results from untreated patients from a Morquio A natural history study (MorCAP).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Maximum voluntary ventilation (MVV) improved up to week 72 and then stabilized; forced vital capacity (FVC) and forced expiratory volume in 1 s (FEV<jats:sub>1<\/jats:sub>) increased continuously over 120 weeks. Mean increases in the modified per\u2010protocol population was 9.2 % for FVC, 8.8 % for FEV<jats:sub>1<\/jats:sub>, and 6.1 % for MVV after 120 weeks. All patients \u226414 years showed respiratory improvements, presumably in part related to growth; however, these were greater in treated patients. For those &gt;14 years, treated patients showed improvements, while deterioration occurred in untreated. Altogether, the improvements were significantly greater (<jats:italic>P<\/jats:italic>\u2009&lt;\u20090.05) in treated patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Long\u2010term ERT is associated with sustained improvements in respiratory function in Morquio A. In younger patients (\u226414 years), some improvement may be ascribed to growth. In older patients, other mechanisms, e.g., decreased glycosaminoglycan storage, are likely involved.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-016-9973-6","type":"journal-article","created":{"date-parts":[[2016,8,23]],"date-time":"2016-08-23T05:34:02Z","timestamp":1471930442000},"page":"839-847","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":29,"title":["Impact of long\u2010term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome"],"prefix":"10.1002","volume":"39","author":[{"given":"Christian J.","family":"Hendriksz","sequence":"first","affiliation":[{"name":"The Mark Holland Metabolic Unit Salford Royal NHS Foundation Trust Ladywell NW2\u2010 2nd Floor Room 112 Salford Manchester M6 8HD UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenneth I.","family":"Berger","sequence":"additional","affiliation":[{"name":"New York University School of Medicine, Andr\u00e9 Cournand Pulmonary Physiology Laboratory Bellevue Hospital New York NY USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rossella","family":"Parini","sequence":"additional","affiliation":[{"name":"Azienda Ospedaliera San Gerardo Monza Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Moeenaldeen D.","family":"AlSayed","sequence":"additional","affiliation":[{"name":"King Faisal Specialist Hospital &amp; Research Center Riyadh Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julian","family":"Raiman","sequence":"additional","affiliation":[{"name":"Hospital for Sick Children Toronto ON Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto","family":"Giugliani","sequence":"additional","affiliation":[{"name":"Med Genet Serv HCPA, Dep Genet UFRGS &amp; INAGEMP Porto Alegre Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John J.","family":"Mitchell","sequence":"additional","affiliation":[{"name":"Montreal Children's Hospital Montreal QC Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara K.","family":"Burton","sequence":"additional","affiliation":[{"name":"Lurie Children's Hospital &amp; NWU Feinberg Chicago IL USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Norberto","family":"Guelbert","sequence":"additional","affiliation":[{"name":"Hospital de Ni\u00f1os de Cordoba Cordoba Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fiona","family":"Stewart","sequence":"additional","affiliation":[{"name":"Belfast City Hospital Belfast NI UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Derralynn A.","family":"Hughes","sequence":"additional","affiliation":[{"name":"Royal Free London NHS Foundation Trust &amp; UC London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Matousek","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc. 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