Compositions and methods for the treatment of fatty acid metabolism disorders
Compositions and methods for inhibiting, treating, and/or preventing fatty acid metabolism disorders, particularly fatty acid oxidation disorders, in a subject are provided.
1. A method for treating a fatty acid oxidation disorder in a subject, said method comprising administering S-nitroso-N-acetyl-cysteine (SNO-NAC) or a pharmaceutically acceptable salt thereof to said subject,
wherein said fatty acid oxidation disorder is very long-chain acyl-coenzyme A dehydrogenase deficiency (VLCADD).
2. The method of claim 1 , wherein the subject has a missense or null mutation within very long-chain acyl-coenzyme A dehydrogenase (VLCAD).
3. The method of claim 2 , wherein the subject has a missense mutation within very long-chain acyl-coenzyme A dehydrogenase (VLCAD).
4. The method of claim 1 , further comprising diagnosing a fatty acid oxidation disorder in said subject prior to administration of said S-nitrosylating agent.
5. The method of claim 4 , wherein said diagnosis comprises:
a) obtaining a biological sample from said subject;
b) determining the enzymatic activity of the very long-chain acyl-coenzyme A dehydrogenase (VLCAD) in said sample; and
c) comparing the amount of VLCAD enzymatic activity determined in step b) to the amount of VLCAD enzymatic activity in a corresponding biological sample from a healthy subject, wherein a decrease in the VLCAD enzymatic activity in the biological sample from the subject compared to the healthy subject is indicative of a fatty acid oxidation disorder in said subject.
6. The method of claim 4 , wherein the diagnosis comprises determining the presence of a mutation in the very long-chain acyl-coenzyme A dehydrogenase (VLCAD) encoding nucleic acid molecule in a biological sample obtained from said subject, wherein the presence of a mutation in the VLCAD encoding nucleic acid molecule is indicative of a fatty acid oxidation disorder in said subject.
7. The method of claim 1 further comprising the administration of at least one other therapeutic agent for the treatment of the fatty acid oxidation disorder.
8. The method of claim 7 , wherein said other therapeutic agent is triheptanoin or bezafibrate.