How should Carnitine Deficiency be treated in infants?
I copy this abstract for you, from the UCSF, Children’s Hospital Central California, 9300 Valley Children’s Place, Madera, CA 93638, USA Abstract Summary: Carnitine deficiency is a secondary complication of many inborn errors of metabolism. Pharmacological treatment with carnitine not only corrects the deficiency, it facilitates removal of accumulating toxic acyl intermediates and the generation of mitochondrial free coenzyme A (CoA). The United States Food and Drug Administration (US FDA) approved the use of carnitine for the treatment of inborn errors of metabolism in 1992. This approval was based on retrospective chart analysis of 90 patients, with 18 in the untreated cohort and 72 in the treated cohort. Efficacy was evaluated on the basis of clinical and biochemical findings. Compelling data included increased excretion of disease-specific acylcarnitine derivatives in a dose–response relationship, decreased levels of metabolites in the blood, and improved clinical status with decre