{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T13:38:20Z","timestamp":1762004300245},"reference-count":29,"publisher":"Wiley","issue":"S3","license":[{"start":{"date-parts":[[2010,10,6]],"date-time":"2010-10-06T00:00:00Z","timestamp":1286323200000},"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":[[2010,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Information regarding the clinical outcome of enzyme replacement therapy (ERT) with recombinant human <jats:italic>N<\/jats:italic>\u2010acetylgalactosamine 4\u2010sulfatase (rhASB) for mucopolysaccharidosis (MPS) VI in Asian patients is limited. We reviewed nine Taiwanese patients with MPS VI (four males and five females; age range 1.4\u201321.1 years) treated with weekly intravenous infusions of rhASB (1.0 mg\/kg) for at least 2 years. We assessed the biochemical and clinical response every 3 months. After 2 years of treatment, seven patients experienced improvement over baseline in the 6\u2010min walk by a mean of 69.3 m (27.3%), and seven also increased the 3\u2010min stair climb by a mean of 47 steps (35.7%). Shoulder range of motion in all patients improved, and Joint Pain and Stiffness Questionnaire scores improved by 0.597 points (30.5%). Four patients had improved pulmonary function [forced expiratory volume in 1 s increased by 0.130 L (26.3%) and forced vital capacity by 0.148 L (27.6%)]. The respiratory disturbance index decreased in the four patients who underwent polysomnography. A mean overall 51% decrease in urinary glycosaminoglycan excretion indicated a satisfactory biochemical response. ERT was well tolerated by all patients. This treatment is thus beneficial and appears to be safe for treatment of MPS VI in Taiwanese patients.<\/jats:p>","DOI":"10.1007\/s10545-010-9212-5","type":"journal-article","created":{"date-parts":[[2010,10,5]],"date-time":"2010-10-05T08:07:14Z","timestamp":1286266034000},"page":"421-427","source":"Crossref","is-referenced-by-count":20,"title":["Enzyme replacement therapy for mucopolysaccharidosis VI\u2014experience in Taiwan"],"prefix":"10.1002","volume":"33","author":[{"given":"Hsiang\u2010Yu","family":"Lin","sequence":"first","affiliation":[{"name":"Department of Pediatrics Mackay Memorial Hospital No.92, Sec. 2 Chung\u2010Shan North Road Taipei 10449 Taiwan"},{"name":"Department of Medical Research Mackay Memorial Hospital Taipei Taiwan"},{"name":"Mackay Medicine, Nursing and Management College Taipei Taiwan"},{"name":"Institute of Clinical Medicine National Yang\u2010Ming University Taipei 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