Activity Provided By:

Relias LLC

Expert Illustrations & Commentaries: Exploring Novel Approaches to Care for Long-Chain Fatty Acid Oxidation Disorder (LC-FAOD)

Overview / Abstract:	Target Audience This educational activity is directed toward pediatricians, primary care physicians, endocrinologists, metabolic dieticians, neurologists, neuromuscular specialists, nurse practitioners, physician assistants, and additional health care professionals involved in the care of patients with metabolic disorders. Activity Overview Long-chain fatty acid oxidation disorders (LC-FAODs) are caused by defects in the metabolic pathway that converts stored long-chain fatty acids into energy within muscles and other cells, leading to a deficiency in mitochondrial energy production during times of physiologic stress and fasting. LC-FAODs cause serious clinical complications, including cardiomyopathy, recurrent episodes of rhabdomyolysis, weakness, hepatic failure, hypoglycemia, and hyperammonemia. These complications have persisted despite decades of dietary therapy, a diet rich in even-carbon, medium- chain triglycerides (MCT), or a combination of both. Premature mortality may reach 60% to 90% in patients with these diseases. In this online, on-demand CME activity, two faculty experts discuss the pathophysiology of LC-FAODs, diagnosis and clinical presentation, and current treatment options for patients with LC-FAODs. With new therapies and multispecialty care, these patients can have longer, higher quality lives. Learning Objectives Upon successful completion of this activity, you should be better prepared to: Determine clinical signs and testing approaches to facilitate LC-FAOD diagnosis Explain the mechanistic rationale that underlies the application of new approved therapy for the treatment of LC-FAOD Evaluate data regarding the safety and efficacy results from clinical trials that evaluate current treatment strategies among patients with LC-FAOD Describe the clinical importance of recent trial findings in the context of evolving treatment standards in the field of LC-FAOD management
Expiration	Jan 31, 2022
Discipline(s)	Physician CME
Format	Online
Credits / Hours	0.75
Accreditation	ACCME
Presenters / Authors / Faculty	Barbara K. Burton, MD Professor of Pediatrics, Division of Genetics, Birth Defects and Metabolism Ann & Robert H. Lurie Children’s Hospital Professor of Pediatrics Northwestern University Feinberg School of Medicine Chicago, IL Jerry Vockley, MD, PhD Professor of Human Genetics University of Pittsburgh Chief of Medical Genetics Director of the Center for Rare Disease Therapy UPMC Children's Hospital of Pittsburgh Cleveland Family Endowed Chair in Pediatric Research Pittsburgh, PA
Sponsors / Supporters / Grant Providers	Physicians’ Education Resource®, LLC, Ultragenyx Pharmaceutical Inc.
Keywords / Search Terms	Relias LLC Endocrinology, Genetics, Genetic Disorders, Metabolic Disorders, Neonatology, Neonatal, Nutrition, Pediatrics Free CE CME

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