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Open Access 01-10-2020 | Rett Syndrome | AdisInsight Report

Triheptanoin: First Approval

Author: Matt Shirley

Published in: Drugs | Issue 15/2020

Abstract

Triheptanoin (Dojolvi™), a synthetic medium-chain triglyceride, is being developed by Ultragenyx Pharmaceutical as a pharmaceutical-grade anaplerotic compound for use in the treatment of inherited metabolic disorders. In June 2020, triheptanoin received its first regulatory approval, in the USA, for use as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). Triheptanoin has also been investigated for use as a treatment in a range of other metabolic disorders or other diseases where energy deficiency is implicated. This article summarizes the milestones in the development of triheptanoin leading to this first regulatory approval for use in the treatment of pediatric and adult patients with LC-FAOD.

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Title: Triheptanoin: First Approval
Author: Matt Shirley
Publication date: 01-10-2020
Publisher: Springer International Publishing
Keyword: Rett Syndrome
Published in: Drugs / Issue 15/2020
Print ISSN: 0012-6667
Electronic ISSN: 1179-1950
DOI: https://doi.org/10.1007/s40265-020-01399-5

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Triheptanoin: First Approval

Abstract

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

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