Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment-A retrospective chart review

This retrospective study represents the largest analysis reported to date of treatment of LC-FAOD with triheptanoin. The data suggest that triheptanoin improves the course of disease by decreasing the incidence and duration of major clinical manifestations and should be the focus of prospective investigations. Significant heterogeneity in the routine clinical care provided to subjects during the periods studied and the natural variation of clinical course of LC-FAODs with time emphasize the need of additional study of the use of triheptanoin.