Long-term galsulfase enzyme replacement therapy in Taiwanese mucopolysaccharidosis VI patients: A case series
详细信息 查看全文
- 作者：Hsiang-Yu Lina ; b ; c ; d ; e ; Chih-Kuang Chuangc ; f ; g ; Chung-Hsing Wangh ; Yin-Hsiu Chieni ; Yu-Mei Wangj ; Fuu-Jen Tsaih ; Yen-Yin Chouk ; Shio Jean Linl ; Hui-Ping Panm ; Dau-Ming Niue ; n ; Wuh-Liang Hwui ; Yu-Yuan Keo ; Shuan-Pei Lina ; b ; c ; p ; 4535lin@gmail.com" class="auth_mail" title="E-mail the corresponding author
- 关键词：MPS ; mucopolysaccharidosis ; ASB ; N-acetylgalactosamine 4-sulfatase ; GAG ; glycosaminoglycan ; ERT ; enzyme replacement therapy ; Galsulfase ; recombinant human N-acetylgalactosamine 4-sulfatase ; Z score ; standard deviation score ; 6MWT ; 6-minute walk test ; 3MSCT ; 3-minute stair climb test ; FVC ; Forced vital capacity ; FEV1 ; forced expiratory volume in 1 ; s ; E/A ; ratio between early and late (atrial) ventricular filling velocity ; LVMI ; left ventricular mass index ; IVSd ; interventricular septum thick
- 刊名：Molecular Genetics and Metabolism Reports
- 出版年：2016
- 出版时间：June 2016
- 年：2016
- 卷：7
- 期：Complete
- 页码：63-69
- 全文大小：532 K
文摘
- •
Nine Taiwanese MPS VI patients treated with weekly intravenous infusions of galsulfase for 6.2 to 11.2 years were reviewed.
- •
Long-term ERT was beneficial and safe for Taiwanese patients with MPS VI.
- •
Long-term ERT reduced urinary GAG and had positive effects on a wide range of clinical functional assessments.