| 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 1) by administering said formulation. |
| 申请公布号 |
WO0238171(A2) |
申请公布日期 |
2002.05.16 |
| 申请号 |
WO2001US47835 |
申请日期 |
2001.11.09 |
| 申请人 |
BIOMARIN PHARMACEUTICAL, INC.;KAKKIS, EMIL, D. |
发明人 |
KAKKIS, EMIL, D. |
| 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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