发明名称 |
TREATMENT OF MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY |
摘要 |
<p>The present invention provides for methods and compositions for treating medium chain acyl-CoA dehydrogenase deficiency. It is based, at least in part, on the discovery that phenylbutyrate can serve as a substrate for medium chain acyl-CoA dehydrogenase. In non-limiting embodiments, phenylbutyrate and/or another source of phenylacetate is administered as a chaperone treatment to patients suffering from medium chain acyl-CoA dehydrogenase deficiency.</p> |
申请公布号 |
EP2838522(A1) |
申请公布日期 |
2015.02.25 |
申请号 |
EP20130778033 |
申请日期 |
2013.04.16 |
申请人 |
UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION |
发明人 |
MOHSEN MOHAMED, AL-WALID;VOCKLEY, GERARD |
分类号 |
A61K31/06;A61K31/12;A61P3/00 |
主分类号 |
A61K31/06 |
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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