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The purpose of this multi-center, international observational study is to help researchers understand the progression of long-chain fatty acid oxidation disorders (LC-FAOD) over time. LC-FAOD are inherited conditions in which the body is unable to break down fats present in normal foods to produce energy. The study will collect information for up to 10 years from adult and pediatric participants with a confirmed diagnosis of LC-FAOD. Although no drug will be provided to participants as part of this study, researchers will assess the long-term safety and effectiveness of Dojolvi® (triheptanoin) in participants who use Dojolvi as part of their treatment for LC-FAOD.
Subject to certain exclusion criteria, this study is accepting people of all ages who have been diagnosed with a LC-FAOD.
Males and Females: All ages
Study participants will visit the study site 8 times for examinations and have 4 telephone follow-up visits during the 10 years of their enrollment.
Duration: 10 years
Ultragenyx Pharmaceutical, Inc.
Study Description at National Institutes of Health
Long-Chain Fatty Acid Oxidation Disorder
Genetic and Genomic Medicine Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Danielle Black, MS
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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