Online Support

The following independent organizations are dedicated to supporting patients and families living with a urea cycle disorder (UCD). If you or a loved one has a UCD, you are familiar with the challenges of managing it day-to-day. Here are community organizations that provide education and support for people with a UCD and other rare conditions:

Independent Organizations for Those Living With UCD
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NUCDF drives innovative research to improve quality of life and leads the search for a cure. A lifeline to UCD patients, families, and medical professionals worldwide seeking information, support, and hope.

http://www.nucdf.org/

CFUCDF has a vision to connect UCD families together, providing support and critical tools to enhance quality of life.

https://ucdfamily.org/

Independent Organizations for Those Living With a Rare Disease
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CCI promotes well-being and decreases isolation for those impacted by chronic illness through support and education.

https://www.thecenterforchronicillness.org/

The mission of Remember the Girls is to raise awareness of the many issues facing female carriers of X-linked genetic disorders; to provide a forum for X-linked females to share their stories, ask questions, provide and receive emotional support; and to advocate for increased attention of the medical community to the physical and emotional issues of females who carry X-linked disorders.

https://www.rememberthegirls.org/

Global Genes is a nonprofit, advocacy organization for patients and families fighting rare and genetic diseases, including cystinosis. Its mission is to eliminate the challenges of rare disease.

https://globalgenes.org/

NORD is a patient advocacy organization dedicated to people with rare diseases and the groups that help them. NORD provides patients and families with advocacy information, assistance programs, and connections to patient organizations.

https://rarediseases.org/

The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.

https://everylifefoundation.org/

ANGEL AID connects rare communities to teach the tools of self-care and how to be listened to without judgment.

https://www.angelaidcares.org/

Our Odyssey’s mission is connecting young adults impacted by a rare or chronic condition with social and emotional support in the hope of improving their quality of life.

https://ourodyssey.org/

Next Step is a nonprofit that shatters limitations and elevates aspirations of young people with serious illness during their transition to adulthood through campferences, mentorship, music therapy, and other tailored programming.

www.nextstepnet.org

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USE and IMPORTANT SAFETY INFORMATION

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

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

  • sleepiness
  • weakness
  • lightheadedness
  • problems with memory
  • worsening of numbness, tingling, or burning in your hands or feet
  • change in taste
  • problems with hearing
  • confusion
  • headache

What is BUPHENYL?

  • BUPHENYL (sodium phenylbutyrate) Tablets is a prescription medicine that can be taken by mouth and BUPHENYL (sodium phenylbutyrate) Powder is a prescription medicine that can be taken by mouth or feeding tube for the long-term management of high blood levels of ammonia (hyperammonemia) caused by a condition called a urea cycle disorder (UCD).
  • BUPHENYL only treats high blood levels of ammonia in patients with carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) and argininosuccinic acid synthetase (AS) deficiencies.
  • BUPHENYL can be used in infants up to 28 days old who have a complete enzyme deficiency, and in patients 1 month of age and older who have a partial enzyme deficiency and have a history of brain damage from high blood levels of ammonia.
  • BUPHENYL must be used along with a low-protein diet and in some cases, dietary supplements.
  • BUPHENYL is not used to treat acute (severe) hyperammonemia, which is a medical emergency.

Do not take BUPHENYL if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience an allergic reaction while taking BUPHENYL.

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

  • have heart failure or decreased kidney function, which may lead to retention of the sodium content of BUPHENYL with potentially serious consequences, such as worsening heart failure, high blood pressure, and swelling.
  • are pregnant or plan to become pregnant. It is not known if BUPHENYL will harm your unborn baby.
  • are breastfeeding or plan to breastfeed. It is not known if BUPHENYL passes into your breast milk. Breastfeeding is not recommended during treatment with BUPHENYL. Talk to your doctor about the best way to feed your baby if you take BUPHENYL.

What are possible side effects of BUPHENYL?

BUPHENYL may cause serious side effects, including:

  • See “What is the most important information I should know about BUPHENYL?”

The most common side effects of BUPHENYL include:

  • decreased appetite
  • body odor
  • bad taste
  • absent or irregular periods in women

These are not all of the possible side effects of BUPHENYL. 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 Patient Package Insert and discuss with your doctor.

USE and IMPORTANT SAFETY INFORMATION

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

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

  • sleepiness
  • weakness
  • lightheadedness
  • problems with memory
  • worsening of numbness, tingling, or burning in your hands or feet
  • change in taste
  • problems with hearing
  • confusion
  • headache

What is BUPHENYL?

  • BUPHENYL (sodium phenylbutyrate) Tablets is a prescription medicine that can be taken by mouth and BUPHENYL (sodium phenylbutyrate) Powder is a prescription medicine that can be taken by mouth or feeding tube for the long-term management of high blood levels of ammonia (hyperammonemia) caused by a condition called a urea cycle disorder (UCD).
  • BUPHENYL only treats high blood levels of ammonia in patients with carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) and argininosuccinic acid synthetase (AS) deficiencies.
  • BUPHENYL can be used in infants up to 28 days old who have a complete enzyme deficiency, and in patients 1 month of age and older who have a partial enzyme deficiency and have a history of brain damage from high blood levels of ammonia.
  • BUPHENYL must be used along with a low-protein diet and in some cases, dietary supplements.
  • BUPHENYL is not used to treat acute (severe) hyperammonemia, which is a medical emergency.

Do not take BUPHENYL if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience an allergic reaction while taking BUPHENYL.

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

  • have heart failure or decreased kidney function, which may lead to retention of the sodium content of BUPHENYL with potentially serious consequences, such as worsening heart failure, high blood pressure, and swelling.
  • are pregnant or plan to become pregnant. It is not known if BUPHENYL will harm your unborn baby.
  • are breastfeeding or plan to breastfeed. It is not known if BUPHENYL passes into your breast milk. Breastfeeding is not recommended during treatment with BUPHENYL. Talk to your doctor about the best way to feed your baby if you take BUPHENYL.

What are possible side effects of BUPHENYL?

BUPHENYL may cause serious side effects, including:

  • See “What is the most important information I should know about BUPHENYL?”

The most common side effects of BUPHENYL include:

  • decreased appetite
  • body odor
  • bad taste
  • absent or irregular periods in women

These are not all of the possible side effects of BUPHENYL. 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 Patient Package Insert and discuss with your doctor.