Triheptanoin in patients with long-chain fatty acid oxidation disorders: clinical experience in Italy
The significant improvement in clinical outcome measures after the administration of triheptanoin highlights that this treatment approach can be more effective than MCT supplementation in patients with LC-FAOD. Triheptanoin was well tolerated and decreased the number of intercurrent catabolic episodes, metabolic decompensation episodes requiring hospitalisation, and the annualised rate and duration of hospitalisations.
Authors
- Elena Procopio (ORCID: https://orcid.org/0000-0002-8534-9951)
- Roberta Taurisano
- Francesco Porta (ORCID: https://orcid.org/0000-0003-3899-4867)
- Vincenza Gragnaniello (ORCID: https://orcid.org/0000-0001-6384-652X)
- Serena Gasperini (ORCID: https://orcid.org/0000-0001-8743-4407)
- A Burlina
- Carlo Dionisi‐Vici (ORCID: https://orcid.org/0000-0002-0007-3379)
- Marco Spada (ORCID: https://orcid.org/0000-0002-8894-350X)
- Arianna Maiorana (ORCID: https://orcid.org/0000-0001-9986-1385)
Institutions
- Azienda Ospedaliera San Gerardo (IT)
- Meyer Children's Hospital (IT)
- Bambino Gesù Children's Hospital (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- University of Turin (IT)
Publication Details
- Journal
- The Italian Journal of Pediatrics/Italian journal of pediatrics
- Published
- 2024-10-07
- DOI
- https://doi.org/10.1186/s13052-024-01782-y
- Citations
- 9
- Primary Topic
- Metabolism and Genetic Disorders
- Type
- article
- Field-Weighted Citation Impact
- 3.43
Funders
- Ultragenyx Pharmaceutical