Future by Design is a group of adults living with cystinosis who’ve come together to pave a brighter future for the teens and adults touched by our rare disease.
We’ve created this Outreach Program to connect individuals and to build relationships within the cystinosis community. Most importantly, we want you to know – you are not alone.
We are here to help make your cystinosis journey easier. Please take a look at our outreach volunteer bios and contact us today! We can chat through text, email, phone calls, and in-person visits based on what’s best for you.
Outreach volunteers can provide access to cystinosis resources, videos and tips from Todd Bradley, and access to Child Life Specialist, Amy Black, equipped with coping techniques, understanding hospital experiences and general life with a rare disease. Check out our Future by Design video.
We want to be a helpful resource for the cystinosis community. Please feel free to reach out with questions or if you just want someone to talk to.
Hi my name is Todd, I was diagnosed with cystinosis at 11 months. I'm now 31, looking forward to my 32nd birthday, and considering myself lucky to be here. I feel strongly about all who are affected by cystinosis, including those with the disease and their family members. But I have some different viewpoints.
Many of those with cystinosis say "cystinosis doesn't define me" but that really frustrates me! I have found my purpose in life through this disease. I have met great and caring people, found a place where I belong, feel comfortable in a place I can make my mark in the sand. You all have inspired me to be grateful, to have courage and confidence, and to know that I have an army behind me. You give me strength to look forward to the next day.
Hello! I’m Emily, 30 years old with cystinosis. I was born in Brazil and then moved to the United States when I was four. Growing up I was into everything as a kid; rollerblading, skateboarding, and all the fun stuff in the 80’s and 90’s. I remember saving up for a PlayStation and till this day I love playing video games! I had my first kidney transplant at ten years old, and it lasted me 10 years. I kept my summers busy by traveling the world. I got married at 18 and was married for 2 years, I had a miracle baby boy, Elijah, who means the world to me. I am so grateful for everyone in my life. I love getting to know people, joking around, listening to music and just enjoying and grasping everything that life has to offer. I am a kid at heart and love new adventures and love being a mommy! Hit me up anytime, 203-531-1733.
Growing up with cystinosis was not easy. As if dealing with the disease itself was not challenging enough, I also had to cope with the multiple adverse side effects that came along with taking the medications. We all know how terrible the Cystagon was to take.
“Living with Cystinosis: A Closer Look”. This video is now available at http://www.youtube.com/watch?
Sigma-Tau Pharmaceuticals Launches Cystaran Today
SIGMA-TAU PHARMACEUTICALS LAUNCHES CYSTARAN™ FOR THE TREATMENT OF CORNEAL CYSTINE CRYSTAL ACCUMULATION IN PATIENTS WITH CYSTINOSIS
GAITHERSBURG, MD, May 1, 2013-Sigma-Tau Pharmaceuticals, Inc. (Sigma-Tau), a part of the Sigma-Tau Group Rare Disease Franchise, announced today the availability of CYSTARAN™ (cysteamine ophthalmic solution) 0.44%, the first and only FDA-approved therapy for the treatment of corneal cystine crystal accumulation in patients with cystinosis. Sigma-Tau developed CYSTARAN in partnership with the National Institutes of Health (NIH) and in cooperation with the Cystinosis Foundation, the Cystinosis Research Foundation, and the Cystinosis Research Network. The U.S. Food and Drug Administration (FDA) approved CYSTARAN in October 2012, and the product has also been granted Orphan Drug status.
The CYSTARAN™ Hotline is available to patients, caregivers and physicians in the U.S.
Order Mon. - Fri., 8:00 a.m. - 8:00 p.m. Eastern
Specialty-trained pharmacists and registered nurses available 24 hours a day, 7 days a week.
CYSTARAN is dispensed solely by Walgreens Specialty Pharmacy.
"The CYSTARAN launch marks an important milestone for Sigma-Tau and further demonstrates our proven ability to develop and deliver novel therapies for patients suffering from a wide range of rare diseases," noted Dave Lemus, Chief Executive Officer of Sigma-Tau. "Sigma-Tau is committed to ensuring comprehensive access for all cystinosis patients with corneal crystal accumulation, and we feel especially privileged to be able to offer this critical new therapy which will make a positive impact on these patients' daily lives."
"For the hundreds of children and adults in the United States who suffer from corneal cystine crystal accumulation as a result of cystinosis, the commercial availability of CYSTARAN provides access to a critically needed therapy," commented Craig B. Langman, MD, the Isaac A. Abt, MD Professor of Kidney Diseases and Head of Kidney Diseases at the Ann & Robert H. Lurie Children's Hospital of Chicago and the Feinberg School of Medicine of Northwestern University. "We need to make sure that physicians and patients managing cystinosis understand the risk of eye complications and are aware of the availability of an FDA-approved therapy with documented safety and effectiveness."
CYSTARAN is available as a sterile ophthalmic solution containing 6.5 mg/mL of cysteamine hydrochloride equivalent to 4.4 mg/mL of cysteamine (0.44%). For full prescribing information for CYSTARAN, see www.cystaran.com.
Safety: The most frequently reported ocular adverse reactions occurring in ≥10% of patients were sensitivity to light, redness, eye pain/irritation, headache, and visual field defects.
Cystinosis, a genetic disease which affects approximately 2,000 individuals worldwide, is a rare, life-threatening condition in which the body accumulates the amino acid cystine (a building block of proteins) within cells. Excess cystine leads to formation of crystals that can build up and damage cells throughout the body. Cystinosis slowly destroys numerous vital organs including the kidneys, liver, eyes, muscles and the brain. Other complications of cystinosis include muscle weakness, diabetes, hypothyroidism, difficulty in swallowing and rickets. Corneal cystine accumulation can lead to ocular complications such as squinting, foreign body sensations, changes in visual acuity, corneal haziness and photophobia (i.e., sensitivity to light). The clinical safety and efficacy of CYSTARAN has been evaluated in controlled clinical trials conducted by the NIH in approximately 300 patients.
About Sigma-Tau Pharmaceuticals, Inc
Sigma-Tau Pharmaceuticals, Inc. is a U.S.-based, wholly owned subsidiary of the Sigma-Tau Group, and is dedicated to the global development and commercialization of medicines for patients with rare diseases. Sigma-Tau Pharmaceuticals, Inc. is based in Gaithersburg, Maryland. Since 1989, the company' s products have been focused on kidney disease, certain genetic disorders and cancers. With more than 7,000 identified rare diseases that affect approximately 30 million patients in the U.S. alone, Sigma-Tau places its considerable scientific resources behind the development and commercialization of compounds that benefit the few. The company has a substantial development program focused on transplant, cancer, inherited genetic disorders, malaria, and other areas of unmet medical need. For more information about the company, visit www.sigmatau.com.
Senior Manager, Patient Affairs
Sigma-Tau Pharmaceuticals, Inc
Senior Vice President
Rx Communications Group
Alana Temple, RN
Clinical Trials Coordinator
Phone: (301) 402-1369
You can view additional photos from the Conference taken by Sarah Dawson Photography at her website here. The password to view the photos is
2018 Scholarship for an Individual Living with Cystinosis
2018 Sierra Woodward Sibling Scholarship
The 2017 recipients are Emily Patterson (CRN Scholarship for an Individual with Cystinosis) and Tyler Morrill(CRN Sierra Woodward Sibling Scholarship). Emily is attending Central Carolina Community College for Early Childhood Education. Tyler is at Miami of Ohio majoring in Kinesiology with plans to become a Physical Therapist.
Congratulations to the past CRN academic scholarship recipients!
Looking for other cystinosis families in your area? With cystinosis being a rare disease, the members of our community are spread all over the world. Sometimes we tend to feel isolated and alone in this journey. If you are interested in networking with other cystinosis families in your area, please complete the form below and someone will contact you.
The Support Groups are an easy way to communicate with the cystinosis community using Yahoo Groups. Yahoo Groups are a way of communicating by email to many people that belong to the same group. CRNSupportGroup and Cystinosisteensupport are the two cystinosis Yahoo Groups affiliated with the Cystinosis Research Network.
For parents, affected adults, caregivers, family, and friends. We also welcome researchers and medical professionals who are interested in cystinosis. This is the place to discuss the various aspects of cystinosis, and how it affects our lives, how we cope with it, vent our frustrations, share our fears, our hopes, and dreams.
For teens with cystinosis and teenage siblings of children and adults with cystinosis. Connect with other teenagers who are dealing with similar issues. The posts include questions, concerns, ideas and supportive sharing.
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