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consider a new option for your LC-FAOD

The first and only FDA-approved treatment for children and adults with long-chain fatty acid oxidation disorders.

Select a type to learn more.

help them reach their goals.

The first and only FDA-approved treatment for children and adults with long-chain fatty acid oxidation disorders.

Select a type to learn more.

CPT I(carnitine palmitoyltransferase I) deficiency

consider a new option for your LC-FAOD

STARTING DOJOLVI

UltraCare® Patient Services is here to help you:

  • Gain access to DOJOLVI (triheptanoin)
  • Understand your insurance coverage
  • Determine your eligibility for financial and patient assistance programs
  • Utilize patient support program resources

Visit UltraCare

A completed Start Form is required for enrollment in UltraCare Patient Services. Talk to your healthcare team.

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TAKING DOJOLVI

Download this step-by-step guide to discuss with your healthcare team how to store, administer, and keep track of DOJOLVI (triheptanoin) doses.

Download the Guide

LC-FAOD defined.

WHAT ARE LC-FAOD?

Long-chain fatty acid oxidation disorders (LC-FAOD) are rare inherited disorders that prevent the body from breaking down long-chain fatty acids into energy during metabolism.

People living with LC-FAOD have difficulty producing enough energy because of their body’s inability to use long-chain fatty acids as an energy source during times of:

  • Fasting
  • Illness
  • Prolonged exercise

Each type of LC-FAOD is named for the specific enzyme that is affected.

Select a type to learn more

CPT I
(carnitine palmitoyltransferase I) deficiency
Cause

Mutation in the CPT1A gene; prevents long-chain fatty acids from being transported into the cells’ mitochondria for breakdown

Estimated Incidence
1:750,000 to 1:2,000,000
Manifestations

Key signs and symptoms manifest from birth to 18 months

CACT
(carnitine-acylcarnitine translocase) deficiency
Cause

Mutation in the SLC25A20 gene; prevents long-chain fatty acids from being transported into the cells’ mitochondria for breakdown

Estimated Incidence
1:750,000 to 1:2,000,000
Manifestations

Key signs and symptoms can present in the neonatal/infant stage of life, while late onset has been reported with less serious symptoms

CPT II
(carnitine palmitoyltransferase II) deficiency
Cause

Mutation in the CPT2 gene; prevents long-chain fatty acids from being transported into the cells’ mitochondria for breakdown

Estimated Incidence
1:750,000 to 1:2,000,000
Manifestations

Key signs and symptoms are distinct in the neonatal/infant stage of life from those in adolescence/young adulthood

VLCAD
(very long-chain acyl-CoA dehydrogenase) deficiency
Cause

Mutation in the ACADVL gene; prevents long-chain fatty acids from being broken down via fatty acid beta-oxidation

Estimated Incidence
1:85,000
Manifestations

Some key signs and symptoms are present throughout life, while some that are present during early childhood are distinct from those that are present during adolescence/adulthood

TFP
(trifunctional protein) deficiency
Cause

Mutations in both the HADHA and HADHB genes, which leads to defects in the entire TFP complex; prevents long-chain fatty acids from being broken down via fatty acid beta-oxidation

Estimated Incidence
1:750,000
Manifestations

Some key signs and symptoms are present throughout life; those that are present in early childhood are similar to those presenting in LCHAD deficiency but are often more serious

LCHAD
(long-chain 3-hydroxy-acyl-CoA dehydrogenase) deficiency
Cause

Mutation in the HADHA gene, which encodes for a subunit of TFP; prevents long-chain fatty acids from being broken down via fatty acid beta-oxidation

Estimated Incidence
1:250,000
Manifestations

Some key signs and symptoms are present throughout life, while some that are present during early childhood are distinct from those that are present during adolescence/adulthood

Meet DOJOLVI (triheptanoin).

WHAT IS DOJOLVI?

DOJOLVI (triheptanoin) is a prescription medicine used to treat long-chain fatty acid oxidation disorders (LC-FAOD) in children and adults.

How does DOJOLVI work?

  • DOJOLVI provides an alternate source of energy. It is a synthetic medium odd-chain 7-carbon triglyceride (C7).
  • DOJOLVI bypasses the enzyme deficiencies that cause LC-FAOD to provide a source of calories and fatty acids that can be converted to energy.

Talk to your healthcare team to learn more about DOJOLVI.

WHAT IS DOJOLVI?

DOJOLVI® (triheptanoin) structure - a synthetic medium odd-chain 7-carbon (C7) triglyceride

WORKING WITH YOUR HEALTHCARE TEAM

All patients treated with DOJOLVI (triheptanoin) should be under the care of clinical specialists knowledgeable in appropriate disease-related dietary management based upon current nutritional recommendations.

These specialists, which may include metabolic geneticists, dietitians, and nurse practitioners, will work together as your healthcare team and:

  • May start you on a low dose of DOJOLVI and slowly increase your dose to help avoid side effects
  • Ensure you are receiving frequent monitoring and nutritional counseling appropriate to your specific needs when taking DOJOLVI

If you are taking another medium-chain triglyceride (MCT) product, stop taking the MCT before starting DOJOLVI.

Learn more about taking DOJOLVI

Stay Informed

Connect with us to get the latest information on LC-FAOD and/or DOJOLVI.

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What is DOJOLVI?

DOJOLVI is a prescription medicine used to treat long-chain fatty acid oxidation disorders (LC-FAOD) in children and adults.

IMPORTANT SAFETY INFORMATION

What are the possible side effects of DOJOLVI?

  • Feeding tube problems. Feeding tubes may not work as well or stop working over time when taking DOJOLVI. Do not use DOJOLVI in feeding tubes made of polyvinyl chloride (PVC), a solid plastic material. Monitor the feeding tube to make sure it is working properly.
  • Intestinal absorption problems in patients with pancreatic insufficiency. If you have pancreatic insufficiency, consult with your healthcare provider as it may affect how well DOJOLVI works.
  • The most common side effects of DOJOLVI are:
    • stomach (abdominal) pain
    • diarrhea
    • vomiting
    • nausea

These are not all the possible side effects of DOJOLVI. Call your healthcare provider for medical advice about side effects. You may report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657 or FDA at 1-800-FDA-1088.

Before taking DOJOLVI, tell your healthcare provider about all of your medical conditions, including if you:

  • are pregnant or plan to become pregnant. It is not known if DOJOLVI will harm your unborn baby. Pregnancy Safety Study: There is a pregnancy safety study for women who take DOJOLVI during pregnancy. The purpose of this study is to collect information about your health and your baby's health. You can talk to your healthcare provider or contact 1-888-756-8657 to enroll in this study or get more information.
  • are breastfeeding or plan to breastfeed. It is not known if DOJOLVI passes into breast milk. Talk to your healthcare provider about the best way to feed your baby if you take DOJOLVI.
  • are taking a pancreatic lipase inhibitor, such as orlistat, as it may affect how well DOJOLVI works.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

How should I take DOJOLVI?

  • See the detailed "Instructions for Use" at the end of the Patient Information Leaflet for instructions about how to mix and take DOJOLVI by mouth in soft foods or drinks or how to mix and give DOJOLVI through feeding tubes.
  • Take DOJOLVI exactly as your healthcare provider tells you.
  • Your healthcare provider may start you on a low dose of DOJOLVI and slowly increase your dose to help avoid side effects. If you are taking another medium chain triglyceride (MCT) product, stop taking the MCT before starting DOJOLVI.

Please see full Prescribing Information, including the Patient Information Leaflet, for additional Important Safety Information.

What is DOJOLVI?

DOJOLVI is a prescription medicine used to treat long-chain fatty acid oxidation disorders (LC-FAOD) in children and adults.

IMPORTANT SAFETY INFORMATION

What are the possible side effects of DOJOLVI?

  • Feeding tube problems. Feeding tubes may not work as well or stop working over time when taking DOJOLVI. Do not use DOJOLVI in feeding tubes made of polyvinyl chloride (PVC), a solid plastic material. Monitor the feeding tube to make sure it is working properly.
  • Intestinal absorption problems in patients with pancreatic insufficiency. If you have pancreatic insufficiency, consult with your healthcare provider as it may affect how well DOJOLVI works.
  • The most common side effects of DOJOLVI are:
    • stomach (abdominal) pain
    • diarrhea
    • vomiting
    • nausea

These are not all the possible side effects of DOJOLVI. Call your healthcare provider for medical advice about side effects. You may report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657 or FDA at 1-800-FDA-1088.

Before taking DOJOLVI, tell your healthcare provider about all of your medical conditions, including if you:

  • are pregnant or plan to become pregnant. It is not known if DOJOLVI will harm your unborn baby. Pregnancy Safety Study: There is a pregnancy safety study for women who take DOJOLVI during pregnancy. The purpose of this study is to collect information about your health and your baby's health. You can talk to your healthcare provider or contact 1-888-756-8657 to enroll in this study or get more information.
  • are breastfeeding or plan to breastfeed. It is not known if DOJOLVI passes into breast milk. Talk to your healthcare provider about the best way to feed your baby if you take DOJOLVI.
  • are taking a pancreatic lipase inhibitor, such as orlistat, as it may affect how well DOJOLVI works.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

How should I take DOJOLVI?

  • See the detailed "Instructions for Use" at the end of the Patient Information Leaflet for instructions about how to mix and take DOJOLVI by mouth in soft foods or drinks or how to mix and give DOJOLVI through feeding tubes.
  • Take DOJOLVI exactly as your healthcare provider tells you.
  • Your healthcare provider may start you on a low dose of DOJOLVI and slowly increase your dose to help avoid side effects. If you are taking another medium chain triglyceride (MCT) product, stop taking the MCT before starting DOJOLVI.

Please see full Prescribing Information, including the Patient Information Leaflet, for additional Important Safety Information.