Latest News
Two Cases of LCFAOD Presenting With Different Types of Cardiomyopathy
People With Rare Diseases Report Facing Stigma, Poor Quality of Life
Researchers Report Fourth Case of Long-Chain 3-Ketoacyl CoA Thiolase Deficiency
Triheptanoin May Be Superior to MCTs for LCFAOD Treatment
Novel Patient-Centered Program Provides Real-World Data for LCFAOD
Resting and Energy Expenditure Equations Useful for LCFAODs
Novel Insights Into Gene Panel Testing for LCFAOD Could Improve Diagnosis
Trifunctional Protein Deficiency Affects Cardiolipin Remodeling
Thermo-Sensitive Proteins Play a Role in the Diagnosis of LCFAOD
Latest Clinical Trials
Features
LCFAOD Patients Face Lifelong Challenges Despite Treatment
Patients with long chain fatty acid oxidation disorder share their stories as experts race to find better treatments for this rare disease.
Using Technology to Boost Ethnic, Geographic Diversity in Clinical Trials
Eliminating geographic and ethnic bias in clinical trials for rare diseases is the goal of Jeeva Informatics, says founder and CEO Harsha K. Rajasimha, PhD.
Two-Day Virtual Conference Will Explore Fatty Acid Oxidation Disorders
The world’s signature academic event for scientists who study fatty acid oxidation disorders (FAODs) is scheduled to take place virtually October 27-28, 2021. The two-day annual conference is hosted by INFORM, the International Network for Fatty Acid Oxidation Research and Management. In previous years, 100 to 200 people have attended the meeting, said Jerry Vockley, MD, PhD,…
Israel Emerges as Global Powerhouse in Rare Disease Research
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
Improving the Lives of Patients With LCFAOD
NOVATO, California — Not long ago, most people with long chain fatty acid oxidation disorder (LCFAOD) had few options for treatment other than snacking on low-fat, high-carbohydrate nutrients every 2 to 6 hours to prevent liver damage. But that has changed in the last 12 months. Triheptanoin (Dojolvi®) on June 30, 2020, became the first…
LCFAOD Clinical Insights
The Relationship Between Inborn Errors of Metabolism and Pediatric Dental Disorders
We shine a spotlight on a lesser-known implication of inborn errors of metabolism: dental effects that require treatment by a dentist.
The Challenges of Living With a Fatty Acid Oxidation Disorder
Patients with fatty acid oxidation disorders often suffer on multiple fronts due to the largely invisible nature of the disease.
The Far-Reaching Impact of Innovative Clinical Trial Designs in Rare Disease
We examine the lasting impact of innovative clinical trial designs for rare diseases on other clinical experiments.
Ocular Manifestations Prevalent in LCHAD Deficiency
Current estimates suggest that about half of patients with long-chain 3-hydroxy-acyl-CoA dehydrogenase deficiency may suffer from retinopathy.
Rare Care Podcast
Perspectives on Long Chain Fatty Oxidation Disorder With Dr. Jerry Vockley
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