Advances in™ Rare Diseases: Fatty Acid Oxidation Disorders – Improving Energy Production Through Alternative Sources

This activity, Advances in™ Rare Diseases: Fatty Acid Oxidation Disorders – Improving Energy Production Through Alternative Sources, developed in Physicians’ Education Resource®, LLC, (PER®) established Advances in™ legacy format, will increase awareness of the presenting signs and symptoms of long-chain fatty acid oxidation disorders (LC-FAODs). Management strategies to improve quality of life (QoL) and emerging new therapies for LC-FAODs will be highlighted, with an emphasis on individualized treatment. Case vignettes throughout will bring important information on this uncommon but highly burdensome metabolic disorder into clinical practice.

You will hear experts discuss ongoing clinical trials and early experience with compassionate use of the novel agent triheptanoin, as they provide their perspectives on how new treatments may impact long-term outcomes of patients with LC-FAOD, and guide participants on potential application to clinical practice.
Learning Objectives

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

Define the pathophysiology and symptoms of LC-FAOD
Identify disease management options to achieve individualized care for patients with LC-FAOD
Apply knowledge of the mechanisms of action and adverse drug reactions of emerging therapies to clinical management of patients with LC-FAOD