This resource list has been compiled by the Cystinosis Research Foundation for cystinosis patients and their families as a quick reference to assist you with your questions concerning the process necessary to obtain medications and prescriptions. This information is intended for general education and should not be construed as advising on diagnosis or treatment of this or any other medical condition.
AAKP Scholarship Program for Cystinosis Patients
The American Association of Kidney Patients (AAKP) just finalized their Cystinosis Scholarship Program. This is a great program for people with cystinosis, ages 5+, to utilize and eliminate possible financial hurdles to participate in curricular or extracurricular programs.
To apply, click this link!
Cystagon® capsules contain cysteamine bitartrate, a cystine depleting agent that lowers the cystine content of cells in patients with cystinosis. CVS ProCare is the sole distributor for Cystagon®.
Contact CVS Specialty Pharmacy:
Hours of Operation (EST): Monday – Friday: 8 a.m. – 6 p.m. The on-call team is available 24/7 for help after hours.
CVS Specialty Pharmacy provides the following services:
Counseling on ordering Cystagon®
Ordering other medications
PROCYSBI® (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules are the first and only cysteamine therapy with 12-hour dosing approved for the management of nephropathic cystinosis in adults and children at least one-year-old.
PROCYSBI helps reduce cystine levels with 2 doses in 24 hours, taken every 12 hours at the same time every day, allowing patients and caregivers to choose consistent times that work for them. Patients should speak with their healthcare professional about PROCYSBI to determine if it’s right for them. More information, including important safety information, can be found at PROCYSBI.com
Horizon By Your Side is a patient support program with dedicated team members who take a personalized approach to meeting the unique needs of patients prescribed Horizon medicines. Your Patient Access Liaison (PAL) leads your dedicated Horizon By Your Side team that is here to help you, from answering questions as you start taking PROCYSBI and through every step along the way. Your PAL is available in person or by email, text, or phone with information and guidance. To learn more, visit HorizonByYourSide.com
To contact a PAL, call 1-855-888-4004
CYSTADROPS® (cysteamine ophthalmic solution) 0.37% is an eye drop solution that was approved in the US in August 2020 and is the first and only FDA-approved cysteamine drop formulation with four times a day dosing. Clinical trials of CYSTADROPS® demonstrated a significant reduction in cystine crystal deposits in all layers of the cornea in people with cystinosis. CYSTADROPS® requires a prescription and is available from Anovo Specialty Pharmacy (866-925-6212).
CYSTARAN® (cysteamine ophthalmic solution) 0.44% is an eyedrop medication used to treat cystine crystal accumulation in the corneas of patients who have cystinosis. Approved by the FDA in 2012, CYSTARAN® was the first ophthalmic cysteamine eyedrop to safely and effectively treat corneal crystal accumulation.
More information, including important safety information, can be found at www.cystaran.com
To order CYSTARAN®, please contact Alliance Rx Walgreens Pharmacy at 1-877-534-9627
NIH: NATIONAL INSTITUTES OF HEALTH
Appointments with Dr. William Gahl
Joy C. Bryant, RN, BSN, CCRC
Research Nurse Specialist
301-443-8690, 102-10460 (page)
National Human Genome Research Institute National Institutes of Health
9000 Rockville Pike
Building 10/Room 3-2551
Bethesda, Maryland 20892
CYSTINOSIS RESEARCH FOUNDATION (CRF)
The Cystinosis Research Foundation is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to ultimately find a cure for this devastating disease. The Cystinosis Research Foundation issues grants for bench and clinical research studies bi-annually in order to accelerate research progress and ensure that cystinosis research is on-going and focused on novel treatments and a cure. The Cystinosis Research Foundation is also dedicated to educating the public and the medical community about cystinosis to ensure early diagnosis and proper treatment.
NORD: NATIONAL ORGANIZATION FOR RARE DISORDERS
NORD (National Organization for Rare Disorders), a 501(c)(3) organization, is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
CheckOrphan is a non-profit organization located in Basel, Switzerland and Santa Cruz, California that is dedicated to rare, orphan and neglected diseases. CheckOrphan offers users an interactive and dynamic platform for all these diseases. This strategy allows visitors to be updated daily on all the latest news and interact with people internationally. This is essential, because due to the nature of these diseases, there is not a large concentration of individuals within any given proximity.