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

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

This activity is supported by an educational grant from Ultragenyx Pharmaceutical Inc.

Credit Available

• Physicians — maximum of 0.75 AMA PRA Category 1 Credit(s)™
• Nurses — 0.75 Contact Hours

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

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

Faculty

Physician Accreditation Statement

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physician Credit Designation

Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of .75 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nursing Credit Designation

Physicians’ Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for .75 Contact Hours.

Disclosures of Conflicts of Interest

In accordance with ACCME guidelines, PER® has identified and resolved all conflicts of interest for faculty, staff, and planners prior to the start of this activity by using a multistep process.

Barbara K. Burton, MD

• Grant Research Support: BioMarin, Denali Therapeutics, Homology Medicines, Sangamo Therapeutics, Takeda, Ultragenyx
• Consultant: Aeglea, BioMarin, Denali Therapeutics, Horizon, Inventiva, JCR Pharmaceuticals, Sanofi Genzyme, Takeda, Ultragenyx
• Speakers Bureau: Alexion, BioMarin, Horizon, Sanofi Genzyme, Takeda

Jerry Vockley, MD, PhD

• Grant Research Support: Alexion, Baebies, Inc, Biomarin, Homology Medicines, Horizon, Kaleido, LogicBio, Mereo, Moderna TX, Inc., Reneo, Sanofi Genzyme, Stealth BioTherapeutics, Ultragenyx Pharmaceuticals; Consultant: Biomarin, LogicBio, Sanofi.

The staff of Physicians’ Education Resource®, LLC (PER®), have no relevant financial relationships with commercial interests to disclose.

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. Complete the activity (including pre- and post-activity assessments).
2. Answer the evaluation questions.
3. Request credit using the drop-down menu.

You may immediately download your certificate.

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

Off-Label Disclosure and Disclaimer

This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or 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 PER® or any company that provided commercial support for this activity.