| 发明名称 |
COMPOSITIONS AND METHODS FOR TREATMENT OF PEROXISOMAL DISORDERS AND LEUKODYSTROPHIES |
| 摘要 |
Compositions and methods for treating, alleviating, and/or preventing one or more symptoms associated with axonal degeneration in individuals in need thereof, such as individuals with peroxisomal disorders and leukodystrophies include one or more poly(amidoamine) dendrimers G1-G10, preferably G4-G6, complexed with therapeutic, prophylactic and/or diagnostic agent in an effective amount to treat, and/or prevent one or more symptoms associated with axonal degeneration are provided. Compositions are particularly suited for targeted delivery of therapeutics to the affected spinal neurons and may contain one or more additional targeting moieties. |
| 申请公布号 |
US2017119899(A1) |
申请公布日期 |
2017.05.04 |
| 申请号 |
US201615339486 |
申请日期 |
2016.10.31 |
| 申请人 |
The Johns Hopkins University ;Kennedy Krieger Institute, Inc. |
发明人 |
Kannan Sujatha;Fatemi Seyed Ali;Rangaramanujam Kannan;Zhang Fan;Turk Bela;Gok Ozgul |
| 分类号 |
A61K47/48;A61K49/00;A61K31/195;A61K31/05;A61K31/573;A61K31/192 |
主分类号 |
A61K47/48 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method for treating a peroxisomal disorder or a leukodystrophy in a subject in need thereof comprising administering to the subject systemically, a pharmaceutically acceptable composition comprising dendrimers complexed, covalently attached or intra-molecularly dispersed or encapsulated with a therapeutic, prophylactic or diagnostic agent for treatment or diagnosis of the disorder. |
| 地址 |
Baltimore MD US |
