{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,24]],"date-time":"2026-03-24T13:28:50Z","timestamp":1774358930283,"version":"3.50.1"},"reference-count":32,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2011,11,30]],"date-time":"2011-11-30T00:00:00Z","timestamp":1322611200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2013,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To outline the design, baseline data, and 5\u2010year follow\u2010up data of patients with mucopolysaccharidosis (MPS) VI enrolled in the Clinical Surveillance Program (CSP), a voluntary, multinational, observational program.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The MPS VI CSP was opened in 2005 to collect, for at least 15 years, observational data from standard clinical and laboratory assessments of patients with MPS VI. Baseline and follow\u2010up data are documented by participating physicians in electronic case report forms.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Between September 2005 and March 2010 the CSP enrolled 132 patients, including 123 who received enzyme replacement therapy (ERT) with galsulfase. Median age at enrolment was 13 years (range 1\u201359). Mean baseline data showed impaired growth, hepatosplenomegaly, and reduced endurance and pulmonary function. The most common findings were heart valve disease (90%), reduced visual acuity (79%), impaired hearing (59%), and hepatosplenomegaly (54%). Follow\u2010up data up to 5 years in patients with pre\u2010 and post\u2010ERT measurements showed a decrease in urinary glycosaminoglycans and increases in height and weight in patients &lt;16 years and suggested reductions in liver and spleen size and improvements in endurance and pulmonary function after ERT was started. Vision, hearing, and cardiac function were unchanged. Safety data were in line with previous reports.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>The CSP represents the largest cross\u2010sectional study of MPS VI to date. This first report provides information on the design and implementation of the program and population statistics for several clinical variables in patients with MPS VI. Data collected over 5 years suggest that ERT provides clinical benefit and is well\u2010tolerated with no new safety concerns.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-011-9410-9","type":"journal-article","created":{"date-parts":[[2011,11,30]],"date-time":"2011-11-30T04:31:50Z","timestamp":1322627510000},"page":"373-384","source":"Crossref","is-referenced-by-count":51,"title":["Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)"],"prefix":"10.1002","volume":"36","author":[{"given":"Christian J.","family":"Hendriksz","sequence":"first","affiliation":[{"name":"Birmingham Children's Hospital Birmingham UK"},{"name":"Department of Clinical Inherited Metabolic Disorders Birmingham Children's Hospital NHS Foundation Trust Steelhouse Lane Birmingham B4 6NH UK"}]},{"given":"Roberto","family":"Giugliani","sequence":"additional","affiliation":[{"name":"Department of Genetics\/UFRGS Medical Genetics Service\/HCPA 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