Future Research Questions
What are the outcomes of patients treated with diets containing various percentages of long chain fat and medium chain fat?
Are outcomes in severe VLCAD improved if the diet contains less than 10% of energy as long chain fat?
Does a diet high in protein improve outcomes?
Do patients treated with triheptanoin have improved outcomes as compared to those treated with MCT?
Is L-carnitine supplementation safe during illness?
Are there benefits of mitochondrial-enhancing supplements in patients with VLCAD and cardiomyopathy?
What is the pathophysiology of rhabdomyolysis and cardiomyopathy in VLCAD?
What are best laboratory measurements to assess effectiveness of diet treatment?
Does a plasma acylcarnitine profile provide useful information to assess effectiveness of the diet and/or suggest need for changes in diet treatment?
What are pregnancy outcomes in VLCAD?
Does the method of skimming breast milk (centrifuge vs non-centrifugal separation) affect the fat content of breast milk and the appropriateness for use in feeding infants with VLCAD?
Does genotype affect phenotype and nutrition recommendations, and if so, how?