Ultragenyx Pharmaceutical (NASDAQ:RARE) commences the U.S. commercial launch of Dojolvi (triheptanoin), approved by the FDA on June 30, for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare chronic and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
The product, designed to provide medium-chain odd-carbon fatty acids as an energy source and metabolic replacement, is the only approved therapy for LC-FAOD in the U.S.
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