DWQA QuestionsCategory: QuestionsKetone supplements and mild cpt2 deficiency
Ryan Doll asked 3 years ago
Is it known how the available exogenic ketone supplements on the marjet might effect someone with a mild case of cpt2 deficiency? Specifically when used in the following ways:

  1. As a daily dietary supplement?
  2. When used in a state of symptom ())muscle breakdown such as myoglobinuria, elevated CPK, and severe skeletal muscle pain) onset?

I am very curious and dont have a local Dr. with enough expertise and understanding of CPT2 deficiency to answer a question like this.

1 Answers
INFORM Physician answered 3 years ago
Ketone suppléments have been proposed as a useful supplement for patients with long chain fatty acid oxidation disorders including CPT2. It provides a usable fuel that bypasses the block. However, it has not been studied in clinical trials and has the downside of adding significant calories to the diet. Thus it needs to be used under the supervision of a knowledgable metabolic physician and dietitian. You should ask you metabolic physician about triheptanoin (Dojolvi), another treatment that can bypass the block in CPT2. It has been shown in clinical trials to improve symptoms in long chain fatty acid oxidations, decreasing muscle symptoms compared to pretreatment. It is a prescription drug so will also need to be managed by your metabolic team. If you tell me where you live, I can give you the closest metabolic treatment center to you. Hope this helps. Dr. Vockley