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Treatment of medium-chain acyl-CoA dehydrogenase deficiency
Treatment of medium-chain acyl-CoA dehydrogenase deficiency
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机译:中链酰基辅酶A脱氢酶缺乏症的治疗
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摘要
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.
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