Ultragenyx Pharmaceutical won FDA approval Tuesday for a therapy that treats a group of rare metabolic disorders that render the body unable to convert a certain type of fat—such as those found in olive oil, fish, and nuts—into energy.
People who have long-chain fatty acid oxidation disorders (LC-FAOD) typically manage the condition with dietary changes or by taking supplements. The treatment from Novato, CA-based Ultragenyx (NASDAQ: RARE) is a purified, synthetic version of a triglyceride, triheptanoin (Dojolvi). It’s designed to provide fatty acids that a patient’s cells can metabolize as an alternative energy source.
LC-FAOD is a genetic condition and… Read more »
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