Ultragenyx Pharma Wins FDA’s First Drug Nod for Rare Metabolic Disorder

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 […]

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