| Enzyme replacement therapy for mucopolysaccharidosis VI-experience in Taiwan. | |
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MedLine Citation:
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PMID: 20924685 Owner: NLM Status: Publisher |
Abstract/OtherAbstract:
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Information regarding the clinical outcome of enzyme replacement therapy (ERT) with recombinant human N-acetylgalactosamine 4-sulfatase (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 1.4-21.1 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-min walk by a mean of 69.3 m (27.3%), and seven also increased the 3-min 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. |
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Authors:
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Hsiang-Yu Lin; Ming-Ren Chen; Chih-Kuang Chuang; Chih-Ping Chen; Dar-Shong Lin; Yin-Hsiu Chien; Yu-Yuan Ke; Fuu-Jen Tsai; Hui-Ping Pan; Shio-Jean Lin; Wuh-Liang Hwu; Dau-Ming Niu; Ni-Chung Lee; Shuan-Pei Lin |
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Publication Detail:
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Type: JOURNAL ARTICLE Date: 2010-10-6 |
Journal Detail:
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Title: Journal of inherited metabolic disease Volume: - ISSN: 1573-2665 ISO Abbreviation: - Publication Date: 2010 Oct |
Date Detail:
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Created Date: 2010-10-21 Completed Date: - Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 7910918 Medline TA: J Inherit Metab Dis Country: - |
Other Details:
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Languages: ENG Pagination: - Citation Subset: - |
Affiliation:
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Department of Pediatrics, Mackay Memorial Hospital, No.92, Sec. 2 Chung-Shan North Road, Taipei, 10449, Taiwan. |
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From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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