The Evolving Treatment and Management Landscape for Patients with Long-Chain Fatty Acid Oxidation Disorders

Target Audience
This activity is designed to meet the educational needs of geneticists, pediatricians, nutritionists, cardiologists, neurologists, endocrinologists, and family medicine.

Program Overview
LC-FAODs (long-chain fatty acid oxidation disorders) are rare, life-threatening, autosomal recessive genetic disorders characterized by acute crises of energy production and chronic energy deficiency. They can lead to severe depletion of glucose in the body and serious complications, which can lead to hospitalizations or early death. Despite early diagnosis through newborn screening and dietary therapy, treatment options have, until recently, been extremely limited. A novel agent has recently been approved that has the potential to transform the treatment of LC-FAODs and improve patient outcomes in this patient population. This activity will address advances in LC-FAOD treatment, as well as aid clinicians in optimizing the care for their patients in concordance with established guidelines and consensus recommendations.

This presentation was held at the 2022 Society for Inborn Metabolic Disorders Annual Meeting in Orlando, FL on April 12, 2022.

Learning Objectives
Upon completion of this activity, participants should be better able to:

Integrate genetic and clinical findings to diagnose LC-FAOD and recognize the potential for variation in presentation and prognosis
Discuss dietary management and major clinical events associated with LC-FAOD
Incorporate newly developed therapies for the treatment of LC-FAOD into optimal patient management decisions
Assess therapies currently being studied that may have the potential to improve patient outcomes