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

Released On
April 22, 2022

Expires On
April 22, 2023

Media Type

Completion Time
55 minutes

Cardiology, Genetics, Hepatology, Neurology, Nutrition, Primary Care

Genetics, Neonatal, Nutrition, Pediatrics

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This activity is provided by EXCEL Continuing Education.


This activity is supported in part by an independent education grant from Ultragenyx Pharmaceutical.

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)
  • Nurses — 1.0 Contact Hours

All other healthcare professionals completing this course will be issued a statement of participation.

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



Jerry Vockley, MD, PhD

University of Pittsburgh
Cleveland Family Endowed Chair in Pediatric Research
Professor of Human Genetics
UMPC Children’s Hospital of Pittsburgh
Chief of Genetic and Genomic Medicine
Director of the Center for Rare Disease Therapy
Pittsburgh, PA


Barbara K. Burton, MD

Professor of Pediatrics (Genetics, Birth Defects, and Metabolism)
Ann & Robert H. Lurie Children’s Hospital
Professor of Pediatrics
Northwestern University Feinberg School of Medicine
Chicago, IL

Physician Accreditation Statement

EXCEL Continuing Education is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.

Physician Credit Designation

EXCEL Continuiing Education designates this enduring activity for a maximum of 1.0 AMA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the enduring activity.

Nursing Continuing Education

EXCEL Continuing Education is accredited by the Florida Board of Nursing to provide nursing contact hours.

Nursing Credit Designation

EXCEL Continuing Education designates this activity for 1 contact hour. Provider # 50-619.

Disclosures of Conflicts of Interest

Jerry Vockley, MD, PhD
  • Consulting Support: AroBioTx, Acer, Satellite Bio, Horizon, Illumina, Jaguar, Applied Therapeutics, Agios, Sanofi, Sangamo
  • Consulting, Travel, and Equity/Ownership Interest: Axcella Health, American Gene
  • Consulting, Travel: LogicBio and BioMarin
Barbara K. Burton, MD
  • Consultant, Speaker, and Investigator: Biomarin and Takeda
  • Consultant, Speaker: Sanofi
  • Consulant: Horizon, Moderna, Aegle, and SIO
  • Consultant, Investigator: JCR Pharma, Denali, Ultragenyx
  • Investigator: Homology Medicine

EXCEL staff and peer reviewer have nothing to disclose.

All relevant financial information has been mitigated.

Instructions for Participation and Credit

There are no fees for participating and receiving CME credit for this enduring activity. To receive CME credit participants must:

  1. Read the CME/CE information and faculty disclosures.
  2. Participate in the online activity.
  3. Submit the evaluation form.

Certificates will be emailed to the participant.

Course Viewing Requirements

Supported Browsers:
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above

Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above

Disclosure of Unlabeled Use

EXCEL Continuing Education requires that faculty participating in any CME activity disclose to the audience when discussing any unlabeled or investigational use of any commercial product or device not yet approved for use in the United States.


This activity is designed for educational purposes. Participants have a responsibility to utilize this information to enhance their professional development to improve patient outcomes. Conclusions drawn by the participants should be derived from careful consideration of all available scientific information. The participant should use his/her clinical judgment, knowledge, experience, and diagnostic decision-making before applying any information, whether provided here or by others, for any professional use.

Contact Information

For CME questions please contact: