Triheptanoin , Rhabdo and CPT2 Adult Onset

Emilio, triheptanoin has been studied in all of the long chain fatty acid oxidation disorders including CPT2 deficiency. The long term clinical trials have shown that it nearly limits episodes of hypoglycemia (typically not a problem in older patients, especially with CPT2), and it reduced episodes related to cardiomyopathy by about two thirds. It did reduce episodes of rhabdomyolysis, but only by about a third. So there is still a ways to go in addressing this important problem. But trying triheptanoin when it becomes clinically available (hopefully later this summer) is worth trying as everyone responds differently. Reviewing your clinical problems with your metabolic physician is always a good idea if you are having significant episodes of rhabdomyolysis because there may be other aspects of your metabolic management that can be optimized. I hope this helps. Dr. Vockley