Bridging Gaps, Optimizing Treatment in Patients With LC-FAODs

Optimizing Triheptanoin Therapy

18 min · 14 nov 2024

Beschrijving

* Clinical safety and efficacy * Initiation and titration * Administration considerations * Individualizing treatment

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4 afleveringen

Clinical Case Vignettes

* Initiating triheptanoin * Dosing modification to improve tolerability

Optimizing Triheptanoin Therapy

* Clinical safety and efficacy * Initiation and titration * Administration considerations * Individualizing treatment

Historical Treatment; Introduction of Triheptanoin

* Dietary modification * MCT supplementation * Triheptanoin mechanism of action

Understanding LC-FAODs

* Autosomal recessive inheritance * Carnitine shuttle * Newborn screening * Clinical features