发明名称 |
Formulation and Method for the Prevention and Treatment of Skeletal Manifestation of Gaucher's Disease |
摘要 |
The invention relates to the prevention and treatment of skeletal manifestation of Gaucher's disease. It relates to a formulation for introducing glucocerebrosidase into the lysosomal compartment of macrophages and/or Gaucher cells in the bone marrow, the formulation comprising glucocerebrosidase encapsulated in erythrocytes. Said erythrocytes may have been subjected to a chemical treatment with an agent so as to promote the targeting of macrophages and/or Gaucher cells in the bone marrow. The treatment may be carried out with a bis(sulphosuccinimidyl) suberate (BS3) solution. The invention also describes drugs and methods for prevention and treatment.
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申请公布号 |
US2010316620(A1) |
申请公布日期 |
2010.12.16 |
申请号 |
US20080867627 |
申请日期 |
2008.06.12 |
申请人 |
BOURGEAUX VANESSA;DUFOUR EMMANUELLE;GODFRIN YANN |
发明人 |
BOURGEAUX VANESSA;DUFOUR EMMANUELLE;GODFRIN YANN |
分类号 |
A61K35/18;A61P19/08 |
主分类号 |
A61K35/18 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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