Current Treatments & Therapies for VLCAD
Daily Management
Day to day life with VLCAD Deficiency means helping the body maintain a steady and reliable source of energy each day. Because the body has difficulty using certain long-chain fats for fuel, treatment focuses on preventing the body from running low on energy and providing alternative energy sources that the body can use more easily.
Most daily care includes regular meals and snacks, often spaced throughout the day to prevent long periods without food. This helps keep blood sugar stable and ensures the body has a continuous supply of fuel. Infants and young children may need to eat more frequently (every 2 to 3 hours), including overnight feeds when recommended by their metabolic team.
Many individuals with VLCAD follow a specialized dietary management plan that limits long-chain fats and includes medium-chain fats, which the body can convert into energy more easily. These fats are often provided through MCT (medium-chain triglyceride) oil or specialized medical formulas recommended by a metabolic dietitian. Some individuals may also be prescribed medications, such as DOJOLVI, that help provide an additional energy source.
Daily management may also include monitoring activity levels, staying well hydrated, and avoiding prolonged fasting, especially during times of growth, exercise, or illness. Families are typically provided with an emergency or “sick day” plan that explains what to do if a child cannot eat well or becomes ill, helping prevent an energy crisis.
With consistent nutrition, routine medical care, and guidance from a metabolic care team, many people with VLCAD can live active and healthy lives. Over time, families become very familiar with their child’s needs and learn how to support stable energy levels each day.
Why Medium-Chain Triglycerides (MCT) Are Needed in the Diet
Medium-chain triglycerides (MCT), such as C8 medical nutrition formulas or oils, are commonly used in VLCAD deficiency because they are easier for the mitochondria to convert into energy. Unlike long-chain fats found in many everyday foods, which require the VLCAD enzyme to be properly broken down for fuel, medium-chain fats bypass part of the blocked metabolic pathway.
Because MCTs follow a different metabolic route, the mitochondria can use them more efficiently as an energy source even when long-chain fat metabolism is impaired. This can help provide a more reliable source of energy, reduce the risk of energy depletion, and better support the muscles, heart, and daily activity levels, while also improving overall stability during periods of growth, illness, or increased energy demand.
MCT Supplementation & Energy Therapies
Living with VLCAD deficiency requires ongoing medical management focused on maintaining safe energy levels, preventing metabolic crises, and improving quality of life. While there is currently no cure for VLCAD or other long-chain fatty acid oxidation disorders (LC-FAOD), medical care has advanced significantly in recent years. Treatment plans are highly individualized and guided by a metabolic geneticist and dietitian who specialize in rare metabolic disorders.
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DOJOLVI is an FDA-approved therapy for long-chain fatty acid oxidation disorders, including VLCAD. It is a specialized oil that provides an alternative fuel source to help the body produce energy more consistently. Research has shown that treatment with triheptanoin can reduce major clinical events such as metabolic crises and improve overall medical management in individuals with LC-FAOD.
Unlike traditional fats, triheptanoin helps supply energy in a way that supports the body’s energy cycle, which may help improve endurance, stability, and tolerance during illness or stress.
It is typically:
Prescribed by a metabolic specialist
Dosed based on caloric needs
Given multiple times per day
Taken orally or sometimes via feeding tube (G-tube)
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Medium-chain triglycerides (MCT), including C8-based medical nutrition formulas such as Lipistart or Monogen, are commonly used in VLCAD dietary management. These fats are easier for the body to convert into energy because they bypass the metabolic block affecting long-chain fats.
Current LC-FAOD treatment guidelines commonly include:
Avoidance of fasting
Adequate energy intake
Long-chain fat restriction
MCT supplementation as a key energy source
Your metabolic team will determine which type of supplementation (C7, C8, or a combination) is appropriate based on your child’s individual needs.
Understanding the Difference Between C7 and C8 MCT in VLCAD Deficiency
For individuals living with VLCAD deficiency and other long-chain fatty acid oxidation disorders (LC-FAOD), specialized fats are often used as part of medical nutrition to provide a safer and more usable energy source. Two of the most commonly discussed options are C8 MCT (medium-chain triglycerides) and C7 (triheptanoin).
While both are used to support energy metabolism, they are not the same and serve slightly different roles in how the body produces energy.
Medical Disclaimer: The information provided on this page is for educational and informational purposes only and is not intended to replace medical advice, diagnosis, or treatment. Individuals and families should always consult their physician, metabolic specialist, and qualified healthcare team before making any changes to their medical care, treatment plan, dietary management, supplements, or participation in clinical trials. Every person with VLCAD deficiency has unique medical needs, and care decisions should be made in partnership with a licensed medical professional familiar with their individual condition.
Interested in learning more about DOJOLVI?
If you are interested in learning more about DOJOLVI® (triheptanoin), talk with your child’s metabolic specialist or care team. Families can also learn more about DOJOLVI by visiting the manufacturer’s website from Ultragenyx Pharmaceutical, which provides educational information about how the therapy works, how it is taken, and what support programs may be available for eligible patients and caregivers. Your metabolic clinic may also be able to connect you with additional resources, educational materials, or patient support services to help guide you through treatment options.
Dietary Management for VLCAD
Dietary management is a cornerstone of living safely with VLCAD deficiency. Because the body cannot properly break down long-chain fats, nutrition plans are carefully structured to provide steady energy while limiting metabolic stress.
What does a “Low-Fat” VLCAD Diet Mean?
A “low-fat” diet for VLCAD deficiency is not the same as a typical low-fat diet used for general health. Instead, it is a medically prescribed nutrition plan specifically designed for individuals with VLCAD and other long-chain fatty acid oxidation disorders (LC-FAOD). Your individualized dietary management plan is designed by a metabolic dietitian and tailored to:
Age
weight
growth stage
activity level
medical history
severity of VLCAD
Typically, dietary plans may include:
Restricted long-chain fat intake
Increased carbohydrates for quick energy
Prescribed MCT or C7 supplementation
Frequent meals and snacks (including overnight feeds in some cases)
Many medical nutrition plans set specific targets for long-chain fat as a percentage of total daily calories while still ensuring essential fatty acids, vitamins, and overall nutritional needs are safely met under medical supervision. Because VLCAD exists on a spectrum and every child’s metabolic needs are unique, families should never adjust fat intake, medical nutrition, or supplementation without guidance from their metabolic specialist and metabolic dietitian.
Check out these dietary management resources to help get you started!
Feeding Support & G-Tubes (When Needed)
Some children living with VLCAD deficiency may require additional feeding support to maintain safe, consistent energy intake, particularly during infancy, illness, growth spurts, or periods of increased metabolic demand. The decision to use a G-tube (gastrostomy tube) is a personal, family-centered medical decision made in collaboration with a metabolic specialist, physician, and care team based on the child’s individual medical needs and energy requirements.
A G-tube may be recommended when:
Frequent feeding is medically necessary to prevent fasting
Overnight nutrition is required to maintain stable energy levels
Oral intake is insufficient during illness or metabolic stress
Preventing energy depletion and metabolic crises becomes a safety priority
G-tubes can support VLCAD management by:
Allowing scheduled overnight feeds to reduce fasting risk
Providing easier administration of medications, MCT, or prescribed medical nutrition
Supporting more consistent and stable energy delivery
Reducing the risk of hypoglycemia and metabolic decompensation during illness or stress
For many families affected by VLCAD and other long-chain fatty acid oxidation disorders (LC-FAOD), a G-tube is considered a proactive medical support tool that enhances safety, energy stability, and quality of life, rather than an indicator of disease severity.
Check out these parent testimonials and resources to learn more about G-tubes!
Living with Very Long-Chain Acyl-CoA Dehydrogenase (VLCAD) deficiency can create significant financial challenges. Even with insurance, the cost of metabolic medications, specialist visits, and emergency hospital care can be difficult for many families managing a rare disease. Fortunately, several programs provide financial assistance for individuals with long-chain fatty acid oxidation disorders, helping reduce the cost of treatment and care.
Financial Assistance for VLCAD Treatment
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Forty-three U.S. states offer Katie Beckett (TEFRA) Medicaid waivers, which allow children with rare diseases or severe disabilities to qualify for Medicaid regardless of their parents’ income. These waivers focus on the child’s medical needs, helping them receive care at home instead of in institutional settings.
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Provides financial help for eligible patients with long-chain fatty acid oxidation disorders, helping cover medication copays, deductibles, and coinsurance.
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Offers support to help patients manage treatment-related costs such as medication copays and insurance expenses.