Treatment of medium-chain acyl-CoA dehydrogenase deficiency
AssigneeUniversity of Pittsburgh—Of the Commonwealth System of Higher Education
InventorsAl-Walid Mohsen Mohamed, Gerard Vockley
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Abstract
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.
Claims12
A method for treating medium chain acyl-CoA dehydrogenase deficiency in a subject suffering therefrom comprising administering, to the subject, an effective amount of phenylbutyrate and/or another source of phenylacetate as a regular therapy regimen.
A method for treating medium chain acyl-CoA dehydrogenase deficiency in a subject suffering therefrom comprising administering, to the subject, an effective amount of phenylbutyrate and/or another source of phenylacetate when the subject is at metabolic risk.
Dependencies are inferred from the claim text.