{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T10:54:04Z","timestamp":1790420044560,"version":"4.1.0"},"reference-count":22,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2019,4,8]],"date-time":"2019-04-08T00:00:00Z","timestamp":1554681600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"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":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2019,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>The impact of galsulfase enzyme replacement therapy in patients with mucopolysaccharidosis (MPS) VI with phenotypes at either end of the disease spectrum was evaluated. The MPS VI Clinical Surveillance Program (CSP) was established to collect long\u2010term observational data from routine clinical and laboratory assessments. A subanalysis of the CSP was performed in patients with pretreatment urinary glycosaminoglycan (uGAG) levels &lt;100\u2009\u03bcg\/mg and \u2265200\u2009\u03bcg\/mg creatinine (low\u2010 and high\u2010uGAG) who had received galsulfase for \u22656 months. uGAG, 6\u2010minute walk test (6MWT), 3\u2010minute stair climb test (3MSCT), pulmonary function measures, height\/growth, cardiac function, and safety were evaluated. Patients with a high\u2010uGAG level at pre\u2010treatment baseline (<jats:italic>N<\/jats:italic>\u2009=\u200968) showed greater impairments in endurance and pulmonary function than those with low\u2010baseline uGAG levels (<jats:italic>N<\/jats:italic>\u2009=\u200939). From pre\u2010treatment baseline, the distance walked on the 6MWT in the low\u2010 and high\u2010uGAG groups increased by a mean (\u00b1SD) of 49 (\u00b1151) meters and 42 (\u00b1165) meters (median follow\u2010up 5.5 and 7.7 years), respectively. The number of stairs\/min climbed in the 3MSCT in the low\u2010 and high\u2010uGAG groups increased by a mean of 18 (\u00b133) and 30 (\u00b145) (median follow\u2010up 2.8 and 3.5 years), respectively. Overall, pulmonary function remained unchanged for both groups. No impact was seen on cardiac function. Galsulfase was generally well tolerated in both groups, with most adverse events being MPS\u2010related complications unrelated to galsulfase. Results of this CSP sub\u2010analysis suggest that galsulfase stabilizes MPS VI in the long\u2010term and has an acceptable safety profile, regardless of baseline disease severity.<\/jats:p>","DOI":"10.1002\/jimd.12079","type":"journal-article","created":{"date-parts":[[2019,3,5]],"date-time":"2019-03-05T04:05:19Z","timestamp":1551758719000},"page":"519-526","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program"],"prefix":"10.1002","volume":"42","author":[{"given":"Paul R.","family":"Harmatz","sequence":"first","affiliation":[{"name":"Department of Gastroenterology, UCSF Benioff Children's Hospital Oakland  Oakland California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina","family":"Lampe","sequence":"additional","affiliation":[{"name":"Department of Child Neurology Justus\u2010Liebig University  Gie\u03b2en Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rossella","family":"Parini","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Fondazione MBBM San Gerardo Hospital  Monza Italy"},{"name":"San Raffaele Telethon Institute for Gene Therapy (SR\u2010TIGET) IRCCS San Raffaele Scientific Institute  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Reena","family":"Sharma","sequence":"additional","affiliation":[{"name":"Department of Endocrinology and Metabolic Medicine, Salford Royal Hospital NHS Foundation Trust  Salford UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elisa L.","family":"Teles","sequence":"additional","affiliation":[{"name":"Department of Metabolic Diseases, S\u00e3o Jo\u00e3o Hospital  Porto Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie","family":"Johnson","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Debbie","family":"Sivam","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zlatko","family":"Sisic","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2019,4,8]]},"reference":[{"key":"e_1_2_10_2_1","first-page":"22","article-title":"Mucopolysaccharidosis VI: pathophysiology, diagnosis and treatment","volume":"21","author":"Harmatz PR","year":"2017","journal-title":"Front Biosci"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-5-5"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-011-9410-9"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410060405"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.3233\/PRM-2010-0104"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-012-9474-1"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.30579"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.2174\/138920111795542688"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2006-2184"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2005.12.014"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2008.04.001"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2004-1023"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.03.018"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-009-9007-8"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/ped.12281"},{"key":"e_1_2_10_17_1","first-page":"1","article-title":"Long\u2010term galsulfase treatment associated with improved survival of patients with mucopolysaccharidosis type VI (Maroteaux\u2010Lamy syndrome): 15 year follow\u2010up from the survey study","volume":"6","author":"Quartel A","year":"2018","journal-title":"JIEMS"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00431-008-0910-z"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.36584"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1186\/1471-2466-13-49"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2017.03.008"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-012-9481-2"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2012.02.005"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fjimd.12079","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jimd.12079","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/jimd.12079","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jimd.12079","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,10]],"date-time":"2023-09-10T08:31:37Z","timestamp":1694334697000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/jimd.12079"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,4,8]]},"references-count":22,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2019,5]]}},"alternative-id":["10.1002\/jimd.12079"],"URL":"https:\/\/doi.org\/10.1002\/jimd.12079","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,4,8]]},"assertion":[{"value":"2018-10-15","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-02-27","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-04-08","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}