Please ensure JavaScript is enabled for purposes of website accessibility What is Ornithine Transcarbamylase (OTC) Deficiency?

What is OTC Deficiency?

OTC-D is a rare, genetic, X-linked condition that can be passed down from both the mother and father but is typically through the mother’s side of the family. If you are an OTC-D carrier, it means you carry the gene that can be passed down to your children. Carriers of OTC-D can experience symptoms that can often times be debilitating and life-threatening.

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X-linked carriers ‘carry’ the OTC-D gene that is caused by a change, or mutation, on the
X chromosome.

OTC-D is a rare genetic condition that could affect other female family members. Identifying those who may be at risk is critical so your family can take necessary steps, such as genetic testing.

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Your OTC-D Family Map

Identify the females in your family who may be a
carrier for OTC-D.

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OTC-D in females: the risks and long-term effects of elevated ammonia levels

The signs and symptoms of OTC-D are often subtle, or can be similar to those of other health conditions. So, paying attention to how you feel is important. Signs and symptoms include:

  • Fatigue or exhaustion
  • Poor appetite
  • Headaches or migraines
  • Forgetfulness
  • Trouble focusing or learning new things
  • Avoiding protein (chicken, beef, fish, nuts, yogurt) or feeling ill after eating foods high in protein
  • Brain fog
  • Mood swings or irritability
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Growing up, I had so many symptoms and was continually told that it was all in my head, that there was nothing wrong with me. To get a diagnosis where we could begin to move forward was a great sense of relief to me.”

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Potential triggers of elevated ammonia can include infection, fever, fasting, surgery, menstruation, pregnancy, and in some cases, high levels of stress.

Testing and Advocating for Your Health

Listen to Juli's perspective on the importance of genetic testing.

  • Transcript

    Getting yourself tested for your future is a great thing if you want to go on and have children. You want to know the chances of passing this on to them. Maybe one of your parents had it and you don’t know if you want to be tested. Just by knowing that, it’ll help you maybe to understand your body more. Maybe some of the symptoms you’ve had are very mild, but they could still be symptoms. I think it would just be good to know for your own peace of mind and your future family’s.

    Growing up, I had so many symptoms and was just continually told that it was all in my head. There was nothing wrong with me. And so, to get a diagnosis where we can begin to move forward and figure this out was a great sense of relief to me.

    Having a good team that you can trust, cause you really are trusting them with your life. It’s very important.

    If you’re having trouble getting through to your doctor, there are so many organizations and resources out there to reach out to. So many other women who are going through what you’re going through that have been dismissed by the medical community.

    That’s why we’re here to educate and to help other women who are on this path. There’s nothing wrong with getting a second opinion. Find a doctor that does listen to you. You need to be with a team, with a group that you trust, that you feel safe and that listens to you and validates your concerns.

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Long-term effects of elevated ammonia levels

OTC-D is the most common type of urea cycle disorder (UCD). For people with UCDs, the liver can’t process ammonia properly and it causes toxic ammonia to build up in the body. Over time, elevated ammonia levels can cause brain damage, memory loss, and other cognitive issues. Managing OTC-D and ammonia levels is important for your overall health.

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Learn more about X-linked conditions

Remember The Girls is a nonprofit organization that provides helpful information for women with X-linked conditions.

Find an OTC-D specialist

Find specialists in your area who understand OTC-D and can help with your management plan.

Managing OTC-D

Don't let your OTC-D symptoms get in the way of life. Learn more about OTC-D and ways to handle all of it.

USE and IMPORTANT SAFETY INFORMATION

What is the most important information I should know about RAVICTI?

RAVICTI may cause serious side effects, including:

Nervous system side effects (Neurotoxicity) Phenylacetate (PAA), a breakdown product of RAVICTI, may cause nervous system side effects. Call your doctor or get medical help right away if you have any of these symptoms while taking RAVICTI:

  • sleepiness
  • lightheadedness
  • change in taste
  • problems with hearing
  • confusion
  • problems with memory
  • worsening of numbness, tingling, or burning in your hands or feet
  • headache
  • feeling very tired (fatigue)
  • nausea
  • vomiting

Your doctor may do blood tests to measure the amount of PAA in your blood during your treatment with RAVICTI.

What is RAVICTI?

  • RAVICTI (glycerol phenylbutyrate) Oral Liquid is a prescription medicine used for long-term management of high blood levels of ammonia (hyperammonemia) caused by a condition called a urea cycle disorder (UCD). RAVICTI should be used if the UCD cannot be managed with a low-protein diet and dietary supplements alone. RAVICTI must be used along with a low-protein diet and in some cases dietary supplements.
  • RAVICTI is not used for the acute treatment of hyperammonemia in people with UCD.
  • It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.

Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you have wheezing, shortness of breath, cough, low blood pressure, flushing, nausea or a rash while taking RAVICTI.

Before taking RAVICTI, tell your doctor about all of your medical conditions, including if you:

  • have liver or kidney problems.
  • have pancreas or bowel (intestine) problems.
  • are pregnant or plan to become pregnant. It is not known if RAVICTI will harm your unborn baby. If you become pregnant during treatment with RAVICTI, call Horizon at 1-866-479-6742 to report the pregnancy.
  • are breastfeeding or plan to breastfeed. It is not known if RAVICTI passes into your breast milk. Breastfeeding is not recommended during treatment with RAVICTI. Talk to your doctor about the best way to feed your baby if you take RAVICTI.

What are possible side effects of RAVICTI?

RAVICTI may cause serious side effects, including:

  • See "What is the most important information I should know about RAVICTI?"

The most common side effects of RAVICTI in adults include:

  • diarrhea
  • gas
  • headache
  • abdomen (stomach) pain
  • vomiting
  • tiredness
  • decreased appetite
  • indigestion or heartburn

The most common side effects of RAVICTI in children 2 years to 17 years of age include:

  • upper abdomen (stomach) pain
  • rash
  • nausea
  • vomiting
  • diarrhea
  • decreased appetite
  • headache

The most common side effects of RAVICTI in children 2 months to less than 2 years of age include:

  • low white blood cell count (neutropenia)
  • vomiting
  • constipation
  • diarrhea
  • fever
  • reduced food intake
  • cough
  • stuffy nose
  • runny nose
  • skin rash
  • small round bumps on the skin

The most common side effects of RAVICTI in children less than 2 months of age include:

  • vomiting
  • rash
  • gastroesophageal reflux
  • increased levels of liver enzymes in the blood
  • decreased appetite and reduced food intake
  • low red blood cell count (anemia)
  • cough
  • loss of too much body fluid (dehydration)
  • too much acid in the blood (acidosis)
  • high blood platelet count (thrombocytosis)
  • low blood platelet count (thrombocytopenia)
  • low blood neutrophil count (type of white blood cell) (neutropenia)
  • high white blood cell count (lymphocytosis)
  • diarrhea
  • gas
  • constipation
  • fever
  • drowsiness (lethargy)
  • irritability
  • agitation

These are not all of the possible side effects of RAVICTI. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

For additional Important Safety Information, click here for the Medication Guide and discuss with your doctor.

USE and IMPORTANT SAFETY INFORMATION

What is the most important information I should know about RAVICTI?

RAVICTI may cause serious side effects, including:

Nervous system side effects (Neurotoxicity) Phenylacetate (PAA), a breakdown product of RAVICTI, may cause nervous system side effects.