发明名称 Methods for treating diseases caused by deficiencies of recombinant alpha-l-iduronidase
摘要 The present invention provides a formulation comprising a pharmaceutical composition comprising a human recombinant alpha-L-iduronidase or biologically active or muteins thereof with a purity of greater than 99%, or in combination with a pharmaceutically acceptable carrier. The present invention further provides methods to treat certain genetic disorders including alpha-L-iduronidase deficiency and mucopolysaccharidosis I (MPS I) by administering said formulation.
申请公布号 AU2899102(A) 申请公布日期 2002.05.21
申请号 AU20020028991 申请日期 2001.11.09
申请人 BIOMARIN PHARMACEUTICAL INC. 发明人 EMIL D. KAKKIS
分类号 C12N15/09;A61K9/08;A61K38/00;A61K38/43;A61K38/47;A61K47/02;A61K47/10;A61K48/00;A61P3/00;A61P19/00;A61P43/00;C12N9/24;C12N9/88;C12N15/56 主分类号 C12N15/09
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