UCD Patient Videos

You may have a rare condition, but you're not alone.

Are you feeling that family and friends don't understand what it's like to live with a urea cycle disorder (UCD)? We can help you connect with patients and families who have been there. If you'd like to talk with another UCD patient or caregiver, sign up to speak with one of our UCD Mentors. Watch the videos below to learn from others living with urea cycle disorders. Learn about starting RAVICTI® (glycerol phenylbutyrate) Oral Liquid, how RAVICTI works, and how to take RAVICTI. There are also real life stories from UCD patients and caregivers and introductions to our UCD Mentors.

RAVICTI is for long-term management of high levels of ammonia in the blood (hyperammonemia) caused by a urea cycle disorder. RAVICTI must be used along with a low-protein diet and, in some cases, dietary supplements. RAVICTI should only be used if the UCD cannot be managed with a low-protein diet and dietary supplements alone. RAVICTI is not used to treat extremely high levels of ammonia in the blood (hyperammonemic crises) in people with UCDs as they may require rapidly acting medication. It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.

Connecting With the Community After UCD Diagnosis

"Just being able to connect with people who understand what you’re going through is amazing." – Amy

Sign up to meet other patients and caregivers who have been there.

Read transcript

Amy:

When I first heard UCD, I had no idea what the doctor was talking about. I had never heard of it. So the doctor said, “don’t Google it because it’s gonna be really scary.” So I did what any mom did, I immediately Googled it, and I thought, oh, my goodness. This is what she has, or this is what she could have?

When you are rare, you wanna know you’re not alone.

So being able to connect with other UCD patients, caregivers who understand what you’re going through is amazing.

Nick:

I’d never heard of it. She explained it to me, but it wasn’t probably until she used to give presentations to the first-year medical students when I first started to understand what it was.

Lauren:

Just ask as many questions as you want. The hard ones, the easy ones, the ones that you think might not be that important. Because every question is important.

David:

When I found out that my son and wife had UCD I felt relief.

I finally, finally knew what we were up against.

But, I had no idea how to treat this. Where to go. And so I felt completely joyous that we had diagnosis and completely lost on how to go about where do I go from here?

But the word is tenacity. You stay at it. Work with your doctor. Work with your insurance. Work with whoever you need. 

Patricia:

When we first got the diagnosis, I felt very alone. And there wasn’t that many resources out there to just find out more, whether it was other patients, other caregivers. Strategies on how to deal with diet and medication. There wasn’t that much out there.

Now there has been like a whole host of resources out there.  And I don’t want somebody to repeat what I have gone through unknowing.

It’s great to get to meet other families who are going through the same struggle that you’re going through.

It’s a relief to know that you’re not alone.

 

APPROVED USES and IMPORTANT SAFETY INFORMATION for 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 to treat extremely high levels of ammonia in the blood (hyperammonemic crisis) in people with UCDs.

It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.

 

IMPORTANT SAFETY INFORMATION

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

RAVICTI may cause serious side effects, including:

Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which 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

 

Who should not take RAVICTI?

Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience any of the following symptoms of an allergic reaction while taking RAVICTI:

  • wheezing
  • shortness of breath
  • cough
  • low blood pressure
  • flushing
  • nausea
  • skin rash

 

What should I tell my doctor before taking RAVICTI?

Tell your doctor about any medical conditions and 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. 
    • Pregnancy Registry: There is a Pregnancy Registry for women who take RAVICTI just before becoming pregnant or who become pregnant during treatment with RAVICTI. The purpose of this registry is to collect information about the health of you and your baby. Talk to your doctor about how you can join the Pregnancy Registry. For more information about this registry, call 1-855-823-2595 or visit www.ucdregistry.com.

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
      • 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.

For additional important safety information, please see the Full Prescribing Information and Medication Guide available at RAVICTI.com and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.

APPROVED USES and IMPORTANT SAFETY INFORMATION for RAVICTI

What is the most important safety information I should know about RAVICTI? RAVICTI may cause serious side effects, including: Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which may cause nervous system side effects.

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 to treat extremely high levels of ammonia in the blood (hyperammonemic crisis) in people with UCDs.

It is not known if RAVICTI is safe and effective for the treatment ofN-acetylglutamate synthase (NAGS) deficiency.

IMPORTANT SAFETY INFORMATION

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

RAVICTI may cause serious side effects, including:

Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which 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

Who should not take RAVICTI?

Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience any of the following symptoms of an allergic reaction while taking RAVICTI:

  • wheezing
  • shortness of breath
  • cough
  • low blood pressure
  • flushing
  • nausea
  • skin rash

What should I tell my doctor before taking RAVICTI?

Tell your doctor about any medical conditions and 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.
        • Pregnancy Registry: There is a Pregnancy Registry for women who take RAVICTI just before becoming pregnant or who become pregnant during treatment with RAVICTI. The purpose of this registry is to collect information about the health of you and your baby. Talk to your doctor about how you can join the Pregnancy Registry. For more information about this registry, call 1-855-823-2595 or visit www.ucdregistry.com.
      • 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
        • 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.

        For additional important safety information, click here for the Full Prescribing Information and Medication Guide and discuss with your doctor.

        You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.

        APPROVED USES and IMPORTANT SAFETY INFORMATION for RAVICTI

        What is the most important safety information I should know about RAVICTI? RAVICTI may cause serious side effects, including: Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which may cause nervous system side effects.

        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 to treat extremely high levels of ammonia in the blood (hyperammonemic crisis) in people with UCDs.

        It is not known if RAVICTI is safe and effective for the treatment ofN-acetylglutamate synthase (NAGS) deficiency.

        IMPORTANT SAFETY INFORMATION

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

        RAVICTI may cause serious side effects, including:

        Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which 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

        Who should not take RAVICTI?

        Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience any of the following symptoms of an allergic reaction while taking RAVICTI:

        • wheezing
        • shortness of breath
        • cough
        • low blood pressure
        • flushing
        • nausea
        • skin rash

        What should I tell my doctor before taking RAVICTI?

        Tell your doctor about any medical conditions and 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.
              • Pregnancy Registry: There is a Pregnancy Registry for women who take RAVICTI just before becoming pregnant or who become pregnant during treatment with RAVICTI. The purpose of this registry is to collect information about the health of you and your baby. Talk to your doctor about how you can join the Pregnancy Registry. For more information about this registry, call 1-855-823-2595 or visit www.ucdregistry.com.
            • 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
              • 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.

              For additional important safety information, click here for the Full Prescribing Information and Medication Guide and discuss with your doctor.

              You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.