Links and Resources

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 insurance, and 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.

living well during covid-19 - Webinar May 28, 2020

An Educational Seminar presented by Dr. Elizabeth Christofferson, PhD, Clinical Psychologist.
Hosted by the Dialysis Patient Citizens and American Psychological Association.   Download the webinar flyer.   
Visit the weblink to sign up or get more information. 

AAKP Cystinosis Scholarship Program

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!

PROCYSBI®

New Announcement
Horizon Therapeutics plc Announces Availability of PROCYSBI® (Cysteamine Bitartrate) Delayed-Release Oral Granules in Packets in the United States.  New dosage form in tear-open packets provides a convenient option for people living with cystinosis. Read full announcement.  View frequently asked questions.

PROCYSBI® 
Patients should talk with their doctor to determine if PROCYSBI® (cysteamine bitartrate) delayed-release capsules is right for them. More information on this medicine is available at www.PROCYSBI.com, including details on Horizon Pharma’s TrancendRare™, a patient-support program created with input from people with nephropathic cystinosis and their caregivers. For the general nephropathic cystinosis community, TranscendRare offers educational information as well as tools to help manage the disease. For eligible people on PROCYSBI, TranscendRare offers access and reimbursement support to help you stay on track with your treatment. Patients must make an appointment with their doctor to determine if PROCYSBI™ is right for them. Please visit the Horizon Pharma website for more information. Horizon Pharma purchased Raptor Pharmaceuticals in 2016.

CYSTAGON®

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 ProCare:

ProCare.AD@cvsprocare.com
888-700-0024
Hours of Operation (EST): Monday – Friday: 8 a.m. – 8 p.m. Saturday: 8 a.m. – 1 p.m.

CVS ProCare provides the following services:

  • Counseling on ordering Cystagon®
  • Ordering other medications
  • Insurance inquiries
  • Trained pharmacist assistance available 24 hours

CYSTARAN™ (EYE DROPS)

CYSTARAN™ is the only FDA-approved ophthalmic therapy for corneal crystals in cystinosis patients. Effective July 31, 2014, Walgreens Specialty Pharmacy will be the sole dispensing pharmacy for CYSTARAN™ call 1-877-534-9627 to speak directly with a Walgreens Specialty Pharmacy team member. To contact Leadiant Biosciences, call Lesli King, Director of Marketing: 301-670-5450 or Lesli.King@leadiant.com. Prescribing information is available here.

NIH: NATIONAL INSTITUTES OF HEALTH

Appointments with Dr. William Gahl

Contact:
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’s vision and guiding principles on which our advocacy initiatives are based:

  • A national awareness and recognition of the challenges faced by people living with rare diseases and the associated costs to society.
  • A nation where people with rare diseases can secure access to diagnostics and therapies that extend and improve their lives.
  • A social, political and financial culture of innovation that supports both the basic and translational research necessary to create diagnostic tests and therapies for all rare disorders.
  • A regulatory environment that encourages development and timely approval of safe and effective diagnostics and treatments for patients with rare diseases.

www.rarediseases.org

MEDICAL RESOURCES

OTHER RESOURCES

RARE: THE GLOBAL GENES PROJECT

CRF was honored to be recognized by Global Genes as a Rare Champion of Hope Awardee for Research and Treatment in 2019.

https://globalgenes.org/rare-champion-of-hope-awards/

New Resource for Teens

Tips for Teens Living with Cystinosis and Kidney Disease

This booklet was developed for tweens and teens who have cystinosis and learn they now have kidney disease and will need dialysis or a transplant. The booklet provides a brief overview of coping with kidney disease, being part of their health care team, the importance of diet, taking medication, and treatment methods. It also touches on feelings related to having another chronic illness, family and friends, and being a teen. The development of this booklet included input from the cystinosis community,  kids currently on dialysis, parents, adults who had kidney disease as children, and nephrologists.

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 insurance, and 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.

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