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.
cystinosis community news
COVID Vaccines and the Cystinosis Community by Paul Grimm, MD, Medical Director, Pediatric Kidney Transplant Program at Stanford University School of Medicine
As the epidemic progresses and we now have the option of Covid vaccines, the question of if or when to take them and vaccine hesitancy has become important. I was asked to contribute a personal message for the cystinosis community. Let me start off by clearly delineating my position.
1) I am firmly committed to vaccines and believe they are a tremendous good for ourselves, our families and our society. Most of my professional colleagues likewise support vaccines.
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.
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.
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® 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:
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)
LEADIANT BIOSCIENCES FEBRUARY 12, 2021
Dear Cystinosis Community:
We wanted to share an update from Leadiant Biosciences regarding the CYSTARAN® (cysteamine ophthalmic solution) 0.44%® eye drops shortage. As you all know, we announced in October 2020 that CYSTARAN went into shortage. The reason why CYSTARAN went into shortage is because Leadiant was working with an independent manufacturer to make CYSTARAN, and that manufacturer is restructuring its business under Chapter 11 of the Bankruptcy Code. The restructuring of this manufacturer during this bankruptcy and various associated issues have affected the continuous production of CYSTARAN.
Fortunately, we have chosen a new manufacturer to begin producing CYSTARAN with a proven track record and expertise in manufacturing. This new partner implemented a new technology that will enable a more consistent and reliable manufacturing process, following the U.S. Food and Drug Administration’s (FDA) standards and requirements. Read More…
LEADIANT BIOSCIENCES NOVEMBER 12, 2020 COMMUNITY UPDATE
Dear Cystinosis Community:
A few weeks ago, we informed you that CYSTARAN® (cysteamine ophthalmic solution) 0.44% eye drops would enter into shortage. We are very sorry for this disruption. Leadiant has a longstanding commitment to you, and we have always strived to be transparent and keep you informed on any news that may affect your treatment. Read more…..
Please know that everyone at Leadiant is committed to have CYSTARAN available to patients as quickly as possible. We have been dedicated to the cystinosis community for more than 20 years, and we are dedicated to working with you through this time. If you have any additional questions regarding this shortage, please call AllianceRx Walgreens Prime at 877-534-9627 or Lesli King at 301-670-5450, Lesli.King@Leadiant.com.
cystadrops (EYE DROPS)
CYSTADROPS (cysteamine ophthalmic solution) 0.37% is a cystine-depleting agent indicated for the treatment of corneal cystine crystal deposits in adults and children with cystinosis.
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’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.
- Gene Tests
- Clinical Genetic Testing
www.cincinnatichildrens.org/service/s/star/genetic-testing/ – Test must be ordered by a physician
- Prevention Genetics
- Kidney Transplants and Donation
- University of California, San Diego
New Resource for Teens
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.