Treatment of medium-chain acyl-CoA dehydrogenase deficiency
View Patent ↗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.
1. 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.
2. The method of claim 1 , where phenylbutyrate is administered as a phenylbutyrate salt.
3. The method of claim 2 , where the salt is sodium phenylbutyrate.
4. The method of claim 1 , where triphenylbutyrylglycerol is administered to the subject as a source of phenylacetate.
5. The method of claim 1 , where a mixture of sodium benzoate and sodium phenylacetate is administered to the subject as a source of phenylacetate.
6. 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.
7. The method of claim 6 , where the subject is at metabolic risk because of an infection.
8. The method of claim 6 , where the subject is at metabolic risk because of a fasting interval.
9. The method of claim 6 where phenylbutyrate is administered as a phenylbutyrate salt.
10. The method of claim 9 , where the salt is sodium phenylbutyrate.
11. The method of claim 6 , where triphenylbutyrylglycerol is administered to the subject as a source of phenylacetate.
12. The method of claim 6 , where a mixture of sodium benzoate and sodium phenylacetate is administered to the subject as a source of phenylacetate.