Summary

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• Triheptanoin (Dojolvi) is indicated as a source of calories and fatty acids for the treatment of adult and pediatric patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).
• Five randomized controlled studies focused on its efficacy in treating LC-FAODs.
• In these studies, triheptanoin demonstrated significant efficacy by reducing major clinical events such as rhabdomyolysis, hypoglycemia, and cardiomyopathy across different patient cohorts.
• The safety profile of triheptanoin was consistent with previous observations; most adverse events were mild to moderate in severity.
• It did not show significant improvement in heart rate during exercise or exercise performance for patients with phosphofructokinase deficiency nor significantly reduce seizure frequency for those with glucose transporter 1 deficiency syndrome.
• The studies highlight that Dojolvi's effectiveness might vary among different patient subtypes within a disorder spectrum.