ReviewDrugs2020
Triheptanoin: First Approval.
Review in Drugs, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 19 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 35 citations in OpenAlex.
- Synthesis and guest inclusion for molecular catcher-based structure determination.Nature protocols · 2026Review
- From clinical practice to mechanistic insights in ketogenic diets for epilepsy.The Lancet. Neurology · 2026Review
- Article
- Anaplerosis by medium-chain fatty acids through complex interplay with glucose and glutamine metabolism.The Journal of biological chemistry · 2025Article
- Anaplerotic filling in heart failure: a review of mechanism and potential therapeutics.Cardiovascular research · 2024Review
- Triheptanoin in patients with long-chain fatty acid oxidation disorders: clinical experience in Italy.Italian journal of pediatrics · 2024Article
- The solute carrier transporters (SLCs) family in nutrient metabolism and ferroptosis.Biomarker research · 2024Review
- Juvenile-Onset Recurrent Rhabdomyolysis Due to Compound Heterozygote Variants in theBrain sciences · 2023Article
- Low Fasting Concentrations of Glucagon in Patients with Very Long-Chain Acyl-CoA Dehydrogenase Deficiency.Metabolites · 2023Article
- Clinical, biochemical and molecular characterization of 12 patients with pyruvate carboxylase deficiency treated with triheptanoin.Molecular genetics and metabolism · 2023Article
- Effects of Triheptanoin on Mitochondrial Respiration and Glycolysis in Cultured Fibroblasts from Neutral Lipid Storage Disease Type M (NLSD-M) Patients.Biomolecules · 2023Article
- A case report of necrotizing enterocolitis in a moderately preterm neonate with LCHADD-A call to focus on the basics while utilizing advanced new therapies.Frontiers in pediatrics · 2023Article
- Population Pharmacokinetics of Heptanoate in Healthy Subjects and Patients With Long-Chain Fatty Acid Oxidation Disorders Treated With Triheptanoin.Clinical pharmacology in drug development · 2022Article
- Metabolic Stress and Mitochondrial Dysfunction in Ataxia-Telangiectasia.Antioxidants (Basel, Switzerland) · 2022Review
- Updates on the ketogenic diet therapy for pediatric epilepsy.Biomedical journal · 2022Review
- Qualitative evaluation of the symptoms and quality of life impacts of long-chain fatty acid oxidation disorders.Therapeutic advances in endocrinology and metabolism · 2022Article
- Triheptanoin - Novel therapeutic approach for the ultra-rare disease mitochondrial malate dehydrogenase deficiency.Molecular genetics and metabolism reports · 2021Article
- An anaplerotic approach to correct the mitochondrial dysfunction in ataxia-telangiectasia (A-T).Molecular metabolism · 2021Article
- A novel therapy for pyridoxine-dependent epilepsy due to biallelic pathogenic variants inTherapeutic advances in rare diseaseArticle
Corrections and comments
- Erratum issued
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
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.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.