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

Released On
December 31, 2018

Expires On
December 31, 2019

Media Type

Completion Time
90 minutes


Fatty Acid Oxidation

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This activity is provided by Physicians' Education Resource®, LLC.

Supported by an educational grant from Ultragenyx Pharmaceutical Inc.

Credit Available

  • Physicians — maximum of 1.50 AMA PRA Category 1 Credit(s)

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

Target Audience

This educational activity is directed toward pediatricians, PCPs, NPs, PAs, and other healthcare professionals involved in the care of patients with LC-FAOD.

Program Overview

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


Jerry Vockley, MD, PhD

University of Pittsburgh
Cleveland Family Endowed Chair in Pediatric Research
Professor of Human Genetics

Children's Hospital of Pittsburgh
Chief of Medical Genetics
Director, Center for Rare Disease Therapy
Pittsburgh, Pennsylvania

Nicola Longo, MD, PhD

Professor and Chief
Division of Medical Genetics
Department of Pediatrics
University of Utah
Salt Lake City, Utah

Accreditation Statement

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians and is a provider approved by the California Board of Registered Nursing (CBRN).

Credit Designation Statement

Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.5 AMA PRA Category 1 Credit(s)™ and for 1.5 contact hours for nurses. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Disclosures of Conflicts of Interest

The following contributors have relevant financial relationships with commercial interests to disclose:

Jerry Vockley, MD, PhD

  • Grant/Research Support: NIH, Reno Pharmaceuticals, Ultragenyx Pharmaceuticals Inc.
  • Investigational/Off-Label Use of Drugs/Devices Disclosure: Triheptanoin (in clinical trials)

Nicola Longo, MD, PhD

  • Grant/Research Support: Ultragenyx Pharmaceuticals Inc. ( support for participation in clinical trials)
  • Investigational/Off-Label Use of Drugs/Devices Disclosure: Triheptanoin (in clinical trials)

Method of Participation

There are no fees to participate in the activity. Participants must review all activity information, including the learning objectives, disclosure statements, and content. To receive credit for participation, participants must complete the post-test (achieving a passing grade) and program evaluation. Certificates can be printed immediately.

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.

Disclaimer/Disclosure of Unlabeled Use

This activity may or may not discuss investigational, unapproved, or off-label uses of drugs. Participants are advised to consult prescribing information for any products discussed. The information provided in this CME activity is for continuing medical education purposes only and is not meant to substitute for the independent medical judgment of a physician relative to the diagnostic, treatment, and management options for a specific patient’s medical condition.

The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of Physicians’ Education Resource®, LLC, or any of the companies that provided commercial support for this activity.