Online Support

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

Independent Organizations for Those Living With Nephropathic Cystinosis

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CRN is an all-volunteer, non-profit organization dedicated to supporting and advocating for research, providing family assistance, and educating the public and medical communities about cystinosis.

https://cystinosis.org/

CRF supports research that aims to improve the quality of life of patients with cystinosis, find better treatments, and ultimately find a cure.

https://www.cystinosisresearch.org/

The Cystinosis Foundation of New Jersey is a non-profit advocacy organization devoted to increasing awareness of cystinosis and funding medical research related to treating and curing those with the condition.

https://cystinosisfoundationofnewjersey.org/

An independent kidney patient organization, AAKP is dedicated to improving the lives and outcomes of kidney patients through education, advocacy, patient engagement, and patient communities.

https://aakp.org/

AKF is a non-profit organization serving the millions of Americans with kidney disease. AKF helps people fight kidney disease and live healthier lives by providing a wide range of programs and services, including prevention activities, educational resources, and cost assistance.

https://www.kidneyfund.org/kidney-disease/other-kidney-conditions/rare-diseases/cystinosis/

DPC is a patient-led, non-profit organization dedicated to improving dialysis patients’ quality of life by advocating for favorable public policy.

https://www.dialysispatients.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/

Global Genes is a non-profit, 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 judgement

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 non-profit 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.

https://www.nextstepnet.org/

USE and IMPORTANT SAFETY INFORMATION 

What is PROCYSBI?

PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a prescription medicine used to treat nephropathic cystinosis in adults and children 1 year of age and older. It is not known if PROCYSBI is safe and effective in children under 1 year of age.

IMPORTANT SAFETY INFORMATION

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

PROCYSBI can cause serious side effects, including:

  • Skin, bone, and joint problems. People treated with high doses of cysteamine bitartrate may develop abnormal changes of their skin and bones, such as stretch marks, bone injuries (such as fractures), bone deformities, and joint problems. Check your skin while taking PROCYSBI. Tell your doctor if you notice any skin changes or problems with your bones or joints. Your doctor will check you for these problems.
  • Skin rash. Skin rash is common with cysteamine bitartrate and may sometimes be severe. Your dose of PROCYSBI may need to be decreased until the rash goes away. If the rash is severe, your doctor may tell you to stop taking PROCYSBI. Tell your doctor right away if you get a skin rash.
  • Stomach and bowel (intestinal) problems. Some people who take other medicines that contain cysteamine bitartrate develop ulcers and bleeding in their stomach or bowel. Tell your doctor right away if you get stomach-area pain, nausea, vomiting, loss of appetite, or vomit blood.
  • Central nervous system symptoms. Some people who take other medicines that contain cysteamine bitartrate develop seizures, depression, and become very sleepy. The medicine may affect how your brain is working (encephalopathy). Tell your doctor right away if you develop any of these symptoms.
  • Low white blood cell count and certain abnormal liver function blood tests. Your doctor should check you for these problems.
  • Benign intracranial hypertension (pseudotumor cerebri) has happened in some people who take immediate-release cysteamine bitartrate. This is a condition where there is high pressure in the fluid around the brain. Your doctor should do eye examinations to find and treat this problem early. Tell your doctor right away if you develop any of the following symptoms while taking PROCYSBI: headache, buzzing or “whooshing” sound in the ear, dizziness, nausea, double vision, blurry vision, loss of vision, pain behind the eye, or pain with eye movement.

Who should not take PROCYSBI?

Do not take PROCYSBI if you are allergic to penicillamine or cysteamine.

What should I tell my doctor before taking PROCYSBI?

Tell your doctor if you have any other medical conditions, including if you:

  • drink alcohol.
  • have a skin rash or bone problems.
  • have or have had stomach or bowel (intestinal) problems including ulcers or bleeding.
  • have a history of seizures, lack of energy, unusual sleepiness, depression, or changes in your ability to think clearly.
  • have liver or blood problems.
  • are pregnant or plan to become pregnant. It is not known if PROCYSBI will harm your unborn baby. Tell your doctor right away if you think that you are pregnant. Talk with your doctor about the benefits and risks of taking PROCYSBI during pregnancy.
  • are breastfeeding or plan to breastfeed. You should not breastfeed during treatment with PROCYSBI. Talk with your doctor about the best way to feed your baby if you take PROCYSBI.

What should I avoid while taking PROCYSBI?

  • Do not drive or operate machinery until you know how PROCYSBI affects you. PROCYSBI can make you sleepy or less alert than normal.
  • Do not drink alcohol if you take PROCYSBI. Drinking alcohol while taking PROCYSBI may change how PROCYSBI works and may cause an increase in the amount of PROCYSBI in your blood that may cause serious side effects.

What are the possible side effects of PROCYSBI?

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

The most common side effects of PROCYSBI include: vomiting, nausea, stomach (abdominal) pain, pink eye, diarrhea, cold, tiredness, flu, headache, problems with body salts or electrolytes, infection of ear, nose or throat, joint pain.

These are not all the possible side effects of PROCYSBI. Call your doctor for medical information about side effects.

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.

For additional important safety information, click here for the Patient Package Insert and discuss with your doctor. 

USE and IMPORTANT SAFETY INFORMATION 

What is PROCYSBI?

PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a prescription medicine used to treat nephropathic cystinosis in adults and children 1 year of age and older. It is not known if PROCYSBI is safe and effective in children under 1 year of age.

IMPORTANT SAFETY INFORMATION

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

PROCYSBI can cause serious side effects, including:

  • Skin, bone, and joint problems. People treated with high doses of cysteamine bitartrate may develop abnormal changes of their skin and bones, such as stretch marks, bone injuries (such as fractures), bone deformities, and joint problems. Check your skin while taking PROCYSBI. Tell your doctor if you notice any skin changes or problems with your bones or joints. Your doctor will check you for these problems.
  • Skin rash. Skin rash is common with cysteamine bitartrate and may sometimes be severe. Your dose of PROCYSBI may need to be decreased until the rash goes away. If the rash is severe, your doctor may tell you to stop taking PROCYSBI. Tell your doctor right away if you get a skin rash.
  • Stomach and bowel (intestinal) problems. Some people who take other medicines that contain cysteamine bitartrate develop ulcers and bleeding in their stomach or bowel. Tell your doctor right away if you get stomach-area pain, nausea, vomiting, loss of appetite, or vomit blood.
  • Central nervous system symptoms. Some people who take other medicines that contain cysteamine bitartrate develop seizures, depression, and become very sleepy. The medicine may affect how your brain is working (encephalopathy). Tell your doctor right away if you develop any of these symptoms.
  • Low white blood cell count and certain abnormal liver function blood tests. Your doctor should check you for these problems.
  • Benign intracranial hypertension (pseudotumor cerebri) has happened in some people who take immediate-release cysteamine bitartrate. This is a condition where there is high pressure in the fluid around the brain. Your doctor should do eye examinations to find and treat this problem early. Tell your doctor right away if you develop any of the following symptoms while taking PROCYSBI: headache, buzzing or “whooshing” sound in the ear, dizziness, nausea, double vision, blurry vision, loss of vision, pain behind the eye, or pain with eye movement.

Who should not take PROCYSBI?

Do not take PROCYSBI if you are allergic to penicillamine or cysteamine.

What should I tell my doctor before taking PROCYSBI?

Tell your doctor if you have any other medical conditions, including if you:

  • drink alcohol.
  • have a skin rash or bone problems.
  • have or have had stomach or bowel (intestinal) problems including ulcers or bleeding.
  • have a history of seizures, lack of energy, unusual sleepiness, depression, or changes in your ability to think clearly.
  • have liver or blood problems.
  • are pregnant or plan to become pregnant. It is not known if PROCYSBI will harm your unborn baby. Tell your doctor right away if you think that you are pregnant. Talk with your doctor about the benefits and risks of taking PROCYSBI during pregnancy.
  • are breastfeeding or plan to breastfeed. You should not breastfeed during treatment with PROCYSBI. Talk with your doctor about the best way to feed your baby if you take PROCYSBI.

What should I avoid while taking PROCYSBI?

  • Do not drive or operate machinery until you know how PROCYSBI affects you. PROCYSBI can make you sleepy or less alert than normal.
  • Do not drink alcohol if you take PROCYSBI. Drinking alcohol while taking PROCYSBI may change how PROCYSBI works and may cause an increase in the amount of PROCYSBI in your blood that may cause serious side effects.

What are the possible side effects of PROCYSBI?

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

The most common side effects of PROCYSBI include: vomiting, nausea, stomach (abdominal) pain, pink eye, diarrhea, cold, tiredness, flu, headache, problems with body salts or electrolytes, infection of ear, nose or throat, joint pain.

These are not all the possible side effects of PROCYSBI. Call your doctor for medical information about side effects.

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.

For additional important safety information, click here for the Patient Package Insert and discuss with your doctor.