Patients with long-chain fatty oxidation disorders (LC-FAODs) have historically been treated with diet modification and even-carbon medium-chain triglyceride (MCT) supplementation. In this 45-minute, case-based, modular activity, Sandra van Calcar, PhD, RD, LD, and Jerry Vockley, MD, PhD, address key topics, including disease presentation, historical treatment, emerging treatment with triheptanoin, and nutritional planning specific to patients with LC-FAODs. Triheptanoin is an odd-carbon MCT designed to bypass the metabolic block in LC-FAODs, recently approved as an alternative treatment option. The faculty discuss the safety and efficacy of triheptanoin and their real-world experience initiating and optimizing it to bridge critical care gaps in patients with LC-FAODs. They briefly orient learners to a downloadable, point-of-care resource for patient education developed as part of this accredited activity.
This content is also availble as a podcast: You may listen here.
| This activity is developed in collaboration with Genetic Metabolic Dietitians International (GMDI) |  |
Course Credit:
0.75 AMA PRA Category 1 CreditsTM
0.75 ANCC Contact Hours
0.75 CA-BRN Contact Hours
0.75 CDR Contact Hours
Dates:
Opens: 2024-11-25
Closes: 2025-11-25
Target Audience:
This activity is intended for metabolic dieticians, nurse practitioners, nurses, and metabolic disease specialists working within Inborn Errors of Metabolism (IEM) clinics in the United States and Canada who care for patients with long chain fatty acid oxidation disorders (LC-FAODs).
This activity is supported by an educational grant from Ultragenyx Pharmaceutical Inc.
Accreditation
The Annenberg Center for Health Sciences at Eisenhower designates this enduring activity for a maximum of 0.75 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Annenberg Center for Health Sciences at Eisenhower designates this enduring activity for a maximum of 0.75 ANCC contact hours.
To receive credit for education contact hours outside of the state of California, please check with your state board of registered nursing for reciprocity.
Registered dietitians (RDs) and dietetic technicians, registered (DTRs) will receive 0.75 hours continuing professional education unit(s) (CPEU) for completion of this program/material.
Additional Content Planners
Vamsi Ahobila, MD (Medical Writer)No significant relationships to disclose.
Joshua Baker, DO, FAAP, FACMG (Peer Reviewer)
Consultant: Ultragenyx
Jessica Kopesky (Peer Reviewer)
Advisory Board: Acer, Horizon, Mead Johnson, Moderna, Nutricia, PTC Therapeutics
Speakers Bureau: BioMarin, Relief Therapeutics
Amber Lambert, MSN, FNP-C, DNP (Nurse Reviewer)
No significant relationships to disclose.
Lindsay Ryan (Peer Reviewer)
Employer: Cycle Pharmaceuticals
Speakers Bureau: Horizon Therapeutics
Danielle Vice (GMDI Education Committee Chair)
No significant relationships to disclose.
Annenberg Center for Health Sciences
Staff at the Annenberg Center for Health Sciences at Eisenhower have no relevant financial relationships to disclose.
All of the financial relationships listed for these individuals have been mitigated.
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Sandra Carol van Calcar, PhD, RD, LD
Associate Professor of Molecular and Medical Genetics
School of Medicine
Oregon Health and Science University
Portland, Oregon -
Jerry Vockley, MD, PhD
University of Pittsburgh
Cleveland Family Endowed Chair in Pediatric Research
Professor of Human Genetics
UPMC Children's Hospital of Pittsburgh
Chief of Genetic and Genomic Medicine
Director of the Center for Rare Disease Therapy
Pittsburgh, Pennsylvania
Presenting Faculty
Downloads
Learning Objectives
- Calculate EER and determine appropriate macronutrient distribution for patients with LC-FAODs, leading to better nutritional support.
- Establish appropriate dosage, titration plans, and administration strategies for triheptanoin in patients with LC-FAODs, considering varying symptom severity, thereby enabling personalized care and optimizing therapy.
- Identify and implement strategies to mitigate GI side effects of triheptanoin therapy, leading to enhanced patient comfort and adherence.
Faculty Disclosures
Sandra Carol van Calcar, PhD, RD, LD
No relationships to disclose.Jerry Vockley, MD, PhD
Consultant: Acer, Agios Pharmaceuticals, Applied Therapeutics, Aro Biotherapeutics, BioMarin Pharmaceutical Inc., Frictionless Solutions, Horizon Therapeutics, Illumina Inc., Jaguar Gene Therapy, LogicBio Therapeutics, Orphan Labs, PTC Therapeutics, Sangamo Therapeutics, Inc., Sanofi, Satellite Bio, Society for Inherited Metabolic Disorders, WebMD Health Corp
Stock: American Gene Technologies International, Inc.