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1.发明授权Precursor N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme 有权前体N-乙酰-4-硫酸酯酶，使用这种酶的生产和纯化这种酶和方法进行治疗的方法
公开(公告)号：EP2327414B1
公开(公告)日：2015-01-07
申请号：EP10011421.4
申请日：2003-11-07
发明人： Qin, Minmin , Henstrand, John M. , Zecherle, Gary N. , Wendt, Dan J. , Chan, Wai-Pan , Chen, Lin , Fitzpatrick, Paul A. , Starr, Christopher M. , Swiedler, Stuart
CPC分类号： B01D15/3804 , A61K38/00 , B01D15/327 , B01D15/3828 , C12N9/16 , C12Y301/06012
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2.发明公开Precursor N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme 有权前体N-乙酰-4-硫酸酯酶，使用这种酶的生产和纯化这种酶和方法进行治疗的方法
公开(公告)号：EP2327414A1
公开(公告)日：2011-06-01
申请号：EP10011421.4
申请日：2003-11-07
发明人： Qin, Minmin , Henstrand, John, M. , Zecherle, Gary, N. , Wendt, Dan, J. , Chan, Wai-Pan , Chen, Lin , Fitzpatrick, Paul, A. , Starr, Christopher, M. , Swiedler, Stuart
CPC分类号： B01D15/3804 , A61K38/00 , B01D15/327 , B01D15/3828 , C12N9/16 , C12Y301/06012
摘要： The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N -acetylgalactosamirie-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N -acetylgalactosamine-4-sulfatase including MPS Vl and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.
摘要翻译： 本发明提供一种高度纯化的重组人前体N-乙酰半乳糖-4-硫酸酯酶和生物学活性突变体，片段和类似物以及药物配方和灰包括高度纯化的重组人前体Ñ-acetylgalactosamirie -4-硫酸酯酶。 因此，本发明提供了用于治疗由人N- -acetylgalactosamine -4-硫酸酯酶包含MPS VI和用于生产和纯化重组前体酶的高度纯化形式的方法的缺陷引起的全部或部分的疾病的方法。
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