{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T15:48:51Z","timestamp":1787500131671,"version":"build-2736575974"},"reference-count":30,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2012,6,5]],"date-time":"2012-06-05T00:00:00Z","timestamp":1338854400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"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>\n                  <jats:p>Characteristic cardiac valve abnormalities and left ventricular hypertrophy are present in untreated patients with mucopolysaccharidosis type VI (MPS VI). Cardiac ultrasound was performed to investigate these findings in subjects during long\u2010term enzyme replacement therapy (ERT) with recombinant human arylsulfatase B (rhASB, rhN\u2010acetylgalactosamine 4\u2010sulfatase, galsulfase, Naglazyme\u00ae). Studies were conducted in 54 subjects before ERT was begun and at specific intervals for up to 96 weeks of weekly infusions of rhASB at 1 mg\/kg during phase 1\/2, phase 2, and phase 3 trials of rhASB. At baseline, mitral and aortic valve obstruction was present and was significantly greater in those \u226512 years of age. Mild mitral and trace aortic regurgitation were present, the former being significantly greater in those &lt;12 years. Left ventricular hypertrophy, with averaged z\u2010scores ranging from 1.6\u20131.9 SD greater than normal, was present for ages both &lt;12 and \u226512 years. After 96 weeks of ERT, ventricular septal hypertrophy regressed in those &lt;12 years. For those \u226512 years, septal hypertrophy was unchanged, and aortic regurgitation increased statistically but not physiologically. Obstructive gradients across mitral and aortic valves remained unchanged. The results suggest that long\u2010term ERT is effective in reducing intraventricular septal hypertrophy and preventing progression of cardiac valve abnormalities when administered to those &lt;12 years of age.<\/jats:p>","DOI":"10.1007\/s10545-012-9481-2","type":"journal-article","created":{"date-parts":[[2012,6,4]],"date-time":"2012-06-04T22:23:43Z","timestamp":1338848623000},"page":"385-394","source":"Crossref","is-referenced-by-count":57,"title":["Enzyme replacement therapy for mucopolysaccharidosis VI: long\u2010term cardiac effects of galsulfase (Naglazyme\n                    <sup>\u00ae<\/sup>\n                    ) therapy"],"prefix":"10.1002","volume":"36","author":[{"given":"E.","family":"Braunlin","sequence":"first","affiliation":[{"name":"Pediatric Cardiology University of Minnesota Minneapolis MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H.","family":"Rosenfeld","sequence":"additional","affiliation":[{"name":"Cardiology Children's Hospital &amp; Research Center Oakland Oakland CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C.","family":"Kampmann","sequence":"additional","affiliation":[{"name":"Department of Congenital Heart Diseases \/Pediatric Cardiology \/GUCH University Medicine, Center for Diseases in Childhood and Adolescence Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J.","family":"Johnson","sequence":"additional","affiliation":[{"name":"Gastroenterology Children's Hospital &amp; Research Center Oakland Oakland CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.","family":"Beck","sequence":"additional","affiliation":[{"name":"Centre for Lysosomal Storage Diseases University Children's Hospital Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R.","family":"Giugliani","sequence":"additional","affiliation":[{"name":"Department of Genetics, UFRGS Porto Alegre RS Brazil"},{"name":"Medical Genetics Service, HCPA Porto Alegre RS Brazil"},{"name":"INAGEMP \u2013 Instituto Nacional de Gen\u00e9tica M\u00e9dica Populacional Porto Alegre RS Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"N.","family":"Guffon","sequence":"additional","affiliation":[{"name":"H\u00f4pital Femme M\u00e8re Enfant Lyon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"D.","family":"Ketteridge","sequence":"additional","affiliation":[{"name":"Metabolic Unit SA Pathology at Women's and Children's Hospital Adelaide Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. 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