“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 2018 recipients are Jack Kitchens and Kathleen Strong (CRN Scholarship for an Individual with Cystinosis) and Emily Ann Bogan and Rhyana Juutilainen (CRN Sierra Woodward Sibling Scholarship). Jack is attending the University of Georgia for Biology while Ms. Strong is studying Nursing at the University of British Columbia. Emily Ann Bogan is a student at Central Carolina Community College and Rhyana Juutilainen is a Criminology major at the University of Nebraska, Omaha.
My name is Jack Kitchens and I am the 2018 recipient of the CRN academic scholarship. I am honored to receive this reward and will make sure to put it to good use. I will use this scholarship to pay for my college books and help further my education. My final goal is to get a degree in Biology and work in a lab. Cystinosis has been a huge part of my life since I was diagnosed at 13 months old. Since then, my family members and many others have done all they can to help me live with cystinosis, and I am extremely thankful for that. The cystinosis community means a lot to me and I hope it continues to help people living with cystinosis. Thank you guys so much for the support, I am forever grateful for it.
One thing that anyone with cystinosis can say is they have been exposed to many, many aspects of health care. Consequently, my health and medical experiences have sparked an intense passion for supporting others. Growing up around nurses, doctors, dieticians, social workers, pharmacists, and technicians has given me insight into many corners of the medical field. However, what has stuck with me the most is the value of the profession of nursing. My relationships with nurses, both on the ward and in outpatient clinics, has had a significant influence on my life outlook. Many of them have become amazing role models for me. My nurses have shown me how a good medical team, and especially a good nurse, can make an enormous, positive difference to someone’s medical story. Because of the care and support I have received from many nurses, it has been my dream and goal in life to provide the same excellent standard of supportive health care to others that I have personally received.
I was fortunate enough to be accepted into the University of British Columbia’s Bachelor of Science in Nursing program and I am extremely grateful for the CRN scholarship which will help finance my education. As I am living on campus and away from home my education expenses are high. This scholarship means so much to me as it is helping me to achieve my career goals and dreams. I am very appreciative.
It is an honor to receive the 2018 Sierra Woodward Academic Scholarship. I am currently attending Central Carolina Community College in Sanford, NC, where I am studying Criminal Justice. Receiving this scholarship will help me achieve my goal of receiving an Associate Degree in Criminal Justice Technology. Following completion at CCCC, it is my dream to find employment in the Criminal Justice field working with addiction and/or at-risk youth. I greatly appreciate the CRN’s recognition and support as I pursue my academic endeavors.
When my first sister was diagnosed with cystinosis in December 2011, I thought my whole world was ending. It was like this diagnosis was this huge brick wall between myself and this idea of the future that I had at the time. However, as the years went by, I started to realize a few things. The first was that cystinosis is sort of like giant brick wall; it’s so big and complicated that it’s hard to explain others who do not face this wall, but that doesn’t mean you are the only one behind the wall and trying to get over or through the wall. I also realized that cystinosis does not stop you from reaching the future that you want. Throughout middle school and high school, I was simply given metaphorical rungs on a latter to help me see past cystinosis. I saw that just because cystinosis puts a strain on my family, that shouldn’t stop me from achieving a future that I want. This scholarship will help get through my first year at the University of Nebraska at Omaha, majoring in Criminology. My hope is one day to work in the FBI or CIA as a Criminologist. Thanks to this scholarship, I am one rung closer to achieving a future that I would not have imagined when my sister was first diagnosed.
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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