Finding the Optimal Treatment Plan for Your Patients With LC-FAODs

Online Course | Specialties: Cardiology, Family Medicine, Neurology, Pediatrics
Released: 6/29/2021
Expires: 6/28/2022
Max Credits: 0.75
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Jerry Vockley, MD, PhD
Professor of Pediatrics and Human Genetics
Cleveland Family Endowed Chair in Pediatric Research
University of Pittsburgh School of Medicine
Director, Center for Rare Disease Therapy
Chief, Medical Genetics
UPMC Children's Hospital of Pittsburgh
Pittsburgh, PA

Activity Planners
Julie Blum, PhD
Director, Clinical Content
Baltimore, MD

Laura Rafferty, ELS
Senior Editorial Manager
Baltimore, MD

Samantha Gordon, MS
Accreditation Manager
Baltimore, MD

Amy Sison
Director of CME
Baltimore, MD

Katherine Kahn
Holyoke, MA

Learning Objectives
Upon completion, participants should be able to:

  • Identify the clinical signs and symptoms of acute metabolic episodes associated with exacerbations of LC-FAODs
  • Individualize treatment plans for patients with LC-FAODs based on their age, energy level, disease severity, and clinical manifestations

Target Audience
This activity is intended for metabolic geneticists, metabolic dietitians, pediatricians, dietitians, registered pharmacists, neurologists, cardiologists, and genetic counselors.

Statement of Need
The management of long-chain fatty acid oxidation disorders (LC-FAODs) is complicated due to their unpredictable and heterogeneous symptoms, different pathophysiologies, and varying disease severity in affected individuals. Until recently, treatment consisted only of lifestyle adjustments, nutritional management, dietary restrictions, and supplementation with medium-chain triglyceride oils, which still left many patients at risk of acute metabolic episodes, emergency medical interventions, hospitalization, and premature death. In 2020, triheptanoin became the first FDA-approved treatment for the management of LC-FAODs, providing another option for patients and underscoring the need for healthcare providers to be familiar with how this new therapy fits into patients’ treatment plans.

Providership Statement
Provided by Med-IQ. 

Accreditation/Designation Statements
Med-IQ is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Med-IQ designates this enduring material for a maximum of 0.75 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurse practitioners, physician assistants, and other healthcare professionals who successfully complete the activity will receive a Statement of Participation indicating the maximum credits available.

Medium/Method of Participation
This CE activity consists of a 0.75-credit online publication. To receive credit, read the introductory CE material, read the publication, and complete the post-survey, evaluation, attestation, and post-test, answering at least 70% of the post-test questions correctly.

Initial Release Date: June 29, 2021
Expiration Date: June 28, 2022
Estimated Time to Complete This Activity: 45 minutes

Disclosure Policy
Med-IQ requires any person in a position to control the content of an educational activity to disclose all relevant financial relationships with any commercial interest. The ACCME defines “relevant financial relationships” as those in any amount occurring within the past 24 months that could create a conflict of interest (COI). Individuals who refuse to disclose will not be permitted to contribute to this CE activity in any way. Med-IQ has policies in place that will identify and resolve COIs prior to this educational activity. Med-IQ also requires faculty to disclose discussions of investigational products or unlabeled/unapproved uses of drugs or devices regulated by the US Food and Drug Administration.

Drug/Product Usage by Faculty
Off-label/unapproved drug uses or products are mentioned within this activity.

Disclosure Statement
The content of this activity has been peer reviewed and has been approved for compliance. The faculty and contributors have indicated the following financial relationships, which have been resolved through an established COI resolution process, and have stated that these reported relationships will not have any impact on their ability to give an unbiased presentation.

Jerry Vockley, MD, PhD
Consulting fees/advisory boards: Agios Pharmaceuticals, American Gene Technologies, Applied Therapeutics, Axcella Health Inc., BioMarin, LogicBio Therapeutics, Inc., MJH Healthcare, Orphan Labs, Sangamo Therapeutics, Inc., Sanofi-aventis U.S. Inc., Synlogic

The writer, peer reviewers, and activity planners have no financial relationships to disclose.

Statement of Evidence-Based Content
Educational activities that assist physicians in carrying out their professional responsibilities more effectively and efficiently are consistent with the ACCME definition of continuing medical education (CME). As an ACCME-accredited provider of CME, it is the policy of Med-IQ to review and ensure that all the content and any recommendations, treatments, and manners of practicing medicine in CME activities are scientifically based, valid, and relevant to the practice of medicine. Med-IQ is responsible for validating the content of the CME activities it provides. Specifically, (1) all recommendations addressing the medical care of patients must be based on evidence that is scientifically sound and recognized as such within the profession; (2) all scientific research referred to, reported, or used in CME in support or justification of a patient care recommendation must conform to generally accepted standards of experimental design, data collection, and analysis.

Med-IQ is not liable for any decision made or action taken in reliance upon the information provided through this activity.

Contact Information
For questions or comments about this activity, please contact Med-IQ. Call (toll-free) 866 858 7434 or email

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The information provided through this activity is for continuing education purposes only and is not meant to substitute for the independent medical judgment of a physician relative to diagnostic and treatment options of a specific patient’s medical condition.

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Complimentary CE
This activity is available free of charge to participants.

Acknowledgment of Commercial Support
This activity is supported by an educational grant from Ultragenyx Pharmaceutical Inc.



Here are the key takeaways from this activity. Deeper insights and evidence, plus an opportunity to receive credit, are available at the "Continue" button below.

  • LC-FAODs are rare, autosomal recessive genetic disorders that cause signs and symptoms related to underlying faulty energy metabolism
  • The most common signs and symptoms are hypoketotic hypoglycemia, muscle weakness, exercise intolerance, rhabdomyolysis, and cardiomyopathy
  • Impaired exercise tolerance, restrictions on physical activity, and frequent hospitalizations adversely affect the quality of life in patients with LC-FAODs and their caregivers
  • Treatment strategies emphasize nutritional restriction of long-chain fatty acids and supplementation with MCTs to bypass the need for long-chain fatty acid oxidation
  • Triheptanoin is a synthetic MCT that was approved by the FDA in 2020 as a source of fatty acids and calories in children and adults with genetically confirmed LC-FAODs
  • Recent studies have shown that triheptanoin use can result in a 50% to 80% decrease in the rate of major clinical events in patients with LC-FAODs, independent from other dietary changes

View reference list.

Click "Continue" to proceed through this activity and/or receive credit. To receive credit and a certificate, you must complete all of the chapters in this activity.

Unless otherwise indicated, photographed subjects who appear within the content of this activity or on artwork associated with this activity are models; they are not actual patients or doctors.

The information provided through this activity is for continuing education purposes only and is not meant to substitute for the independent medical judgment of a physician relative to diagnostic and treatment options of a specific patient’s medical condition.

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