• 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. (cystinosis.org)
  • 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. (cystinosis.org)
  • 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. (cystinosis.org)
  • Other complications of cystinosis include muscle weakness, diabetes, hypothyroidism, difficulty in swallowing and rickets. (cystinosis.org)
  • Cystinosis was the first documented genetic disease belonging to the group of lysosomal storage disease disorders. (wikipedia.org)
  • 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. (cystinosis.org)
  • Cystinosis is a rare, genetic metabolic disease that causes the amino acid cystine to accumulate in various organs of the body. (cystinosis.org)
  • The Company is focused on the development of its gene therapy candidate, AVR‑RD‑01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, Cystinosis and Pompe disease. (avrobio.com)
  • Leadiant Biosciences, Inc. is pleased to share with the cystinosis community that Cystaran® (cysteamine ophthalmic solution) 0.44% will be available for order early next week. (cystinosis.org)
  • 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. (cystinosis.org)
  • Lucky AW, Howley PM, Megyesi K, Spielberg SP, Schulman JD (1977) Endocrine studies in cystinosis: compensated primary hypothyroidism. (springer.com)
  • Receive your FREE copy of the Cystinosis comic book series by completing the form below. (cystinosis.org)
  • Without specific treatment, children living with Cystinosis have a significantly compromised life span and quality of life. (cystinosis.org)
  • Schneider JA, Wong V, Bradley K, Seegmiler JE (1968) Biochemical comparisons of the adult and childhood forms of cystinosis. (springer.com)
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