TranscendRare™, a HorizonCares™ program, is a patient-support program created with input from people with nephropathic cystinosis and caregivers who understand the challenges and successes you may experience. TranscendRare provides valuable services to support you on your journey to manage cystinosis.

For the general nephropathic cystinosis community, TranscendRare offers important information about nephropathic cystinosis, as well as valuable tools to help you better manage the challenges you may face along the way.

For eligible people on PROCYSBI® (cysteamine bitartrate) delayed-release capsules, TranscendRare offers access and reimbursement support to help you stay on track with your treatment.

To get started with TranscendRare, call 855-888-4004

TranscendRare: Supporting you every step of the way

Services and support offered through TranscendRare include:

  • Reimbursement assistance for insured patients
    • TranscendRare can advocate on your behalf to help ensure coverage for PROCYSBI and to resolve contested claims
    • Copay assistance is available for eligible patients and may reduce the out-of-pocket costs for PROCYSBI
  • Reimbursement assistance for underinsured patients
    • TranscendRare helps find available alternate or supplemental insurance
    • Qualified patients may be eligible for financial assistance to help cover premiums
    • If no insurance coverage can be secured, the TranscendRare program may provide PROCYSBI to eligible patients
  • Travel assistance
  • White blood cell (WBC) cystine testing assistance
    • TranscendRare also offers reimbursement assistance to cover the cost associated with WBC cystine testing for eligible patients, when those patients do not receive reimbursement from their insurance providers

Patient Access Managers

Your Patient Access Manager (PAM) is available to support you as a personal resource. Your dedicated PAM will assist you in getting off to a great start with PROCYSBI and help you along the way. Your PAM can help in the following areas:

  • Insurance*
    • Review and discuss insurance challenges
    • Troubleshoot problems with insurance payments and prescription refills
    • Connect you with copay assistance or co-insurance programs
  • Travel and financial assistance*
    • Discuss options for travel to see your cystinosis doctor
    • Discuss support for out-of-pocket costs for white blood cell (WBC) cystine testing
  • Life transitions
    • Help you stay on track when you are going through life changes, such as:
      • Moving to a new city
      • Changing jobs or going off to college
      • Switching schools or teachers
      • Transitioning to adult care
      • Changing doctors
  • Linking you to the cystinosis community
    • Connect you with other people living with cystinosis
    • Inform you of cystinosis events in your area
    • Introduce you to cystinosis advocacy groups

*Assistance may include the cost of transportation, lodging, or meals. Service provided by the PAM are for eligible and/or qualified patients only. Please contact your PAM to determine eligibility.

To connect with your PAM, just call 844-830-CARE (2273)
PAMs are also available via e-mail or text, or in person

Accredo Specialty Pharmacy

PROCYSBI® (cysteamine bitartrate) delayed-release capsules is only available through Accredo, a specialty pharmacy. TranscendRare and Accredo work together to make sure eligible patients have access to PROCYSBI.

TranscendRare™ Program**
insurance Works directly with your insurance
you-and-doctor Keeps in touch with you and your doctor
phone-chat Refers you to other options for financial support
Accredo Specialty Pharmacy
woman Provides you with a dedicated contact person
calendar Reminds you when it’s time to schedule and ship refills
truck Delivers to your door
you-and-doctor Obtain a PROCYSBI prescription from your doctor
keep-in-touch Return calls and keep in touch with TranscendRare
phone" Return Accredo Specialty Pharmacy’s phone calls

**TranscendRare provides personalized support for eligible patients and caregivers in the United States who are prescribed PROCYSBI. Call for eligibility for assistance with the WBC testing and travel related to cystinosis care.

Resources & downloads

Whether you’re a patient with cystinosis or a caretaker of one, dealing with this disease can seem overwhelming at times. But remember that you’re not alone and that help is available. For additional resources, including support and advice for parents, siblings, and students, see the links below.

Educational and social support

Children with cystinosis typically display normal intelligence and average school performance and should be encouraged to pursue their educational interests. Nevertheless, some children may have learning problems related to visual processing, math, and spelling.1 In addition, problems with attention, social interaction, and academic performance can occur. However, with the right support, there’s no reason that children with cystinosis need to limit themselves.

Individualized education programs and 504 plans

A 504 plan can help students with chronic conditions receive modifications and accommodations that will ensure their academic success in a general education setting. For example, a student with cystinosis who has a 504 plan can arrange to have extra time for tests and homework assignments and permission to wear sunglasses due to light sensitivity, among other helpful accommodations.

An individualized education program (IEP) is a written document that guides the education of a student with a disability. Most students between 3 and 21 years of age who are classified with a disability according to the Individuals with Disabilities Education Act of 2004 (IDEA) will have a current IEP.3 Because each IEP is designed for a single student, it is truly an individualized document.4

It is not uncommon for a child with cystinosis to have a 504 plan or IEP in place. An IEP can help a student with cystinosis get additional support to aid with learning challenges. To learn more about 504 plans and IEPs, including how to write one for your child with cystinosis, see the links below.

PROCYSBI downloads

Download helpful tools and information about PROCYSBI.

Cystinosis organizations, advocacy groups, and other resources

Visit these helpful websites for additional information and support for living with cystinosis.

What is PROCYSBI (Pro-CIS-bee)?

PROCYSBI® (cysteamine bitartrate) delayed-release capsules is a prescription medicine used to treat a medical condition called 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.

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?

Before you take PROCYSBI, tell your doctor 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
  • have any other medical conditions
  • 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.

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

Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

What should I avoid while taking PROCYSBI?

Do not drive or operate heavy 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? 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. These changes may include 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. Tell your doctor right away if you get a skin rash. 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.
  • Stomach and bowel (intestinal) 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.

The most common side effects with PROCYSBI include vomiting, nausea, stomach (abdominal) pain, breath odor, diarrhea, skin odor, tiredness, skin rash, headache, and problems with body salts or electrolytes.

Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all of the possible side effects of PROCYSBI. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-FDA-1088.

Issued: 12/2017                    Piece number: P-PYB-00032                    Derived from Patient Information approved 12/2017