Erik Harris, Executive Vice President and Chief Commercial Officer at Ultragenyx Pharmaceutical Inc. discusses the recent approval of triheptanoin (Dojolvi) and gives an overview of long-chain fatty acid oxidation disorders (LC-FAOD). 

LC-FAOD is a group of rare autosomal recessive genetic disorders characterized by the body’s inability to convert long-chain fatty acids into energy. This causes severe depletion of glucose in the body, which can lead to serious complications, hospitalizations, and even early death. LC-FAOD affects 2,000 to 3,500 children and adults across the U.S.

Recently, triheptanoin was announced as the first and only FDA approved treatment for all six types of LC-FAODs. Triheptanoin works by providing medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD. Prior to the approval of triheptanoin, the typical treatment for LC-FAOD included low-fat/high-carbohydrate diets, supplemental carnitine, and even-carbon medium-chain triglyceride (MCT) oil.

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