DWQA QuestionsMore Mitochondria and Retinopathy in LCHADD
Stephanie Harry asked 5 years ago
Is there still any concern that if we boost the number of mitochondria in patients with LCHADD that it could increase the amount of toxic by-products, and thus have a negative effect on their eyes?

1 Answers
INFORM Physician answered 5 years ago
It’s amazing how much you all know about your diseases! While there is no way to know the answer to this question directly, I think it is unlikely to be a problem. In LCHAD deficiency, boosting the amount of enzyme is more likely to decrease the metabolite load than increase it. While our studies are still in progress, we don’t see much of an increase in the number of mitochondria in FAOD cells when we treat with transcriptional activators, even while we see an increase in enzyme. Hope this helps. Dr. Vockley