SOURCE: Cystinosis Research Foundation

Cystinosis Research Foundation

January 23, 2012 12:12 ET

Leading Cystinosis Researchers to Meet in Irvine, Calif., for Third International Symposium on Rare Disease

IRVINE, CA--(Marketwire - Jan 23, 2012) - The third International Cystinosis Research Symposium for scientists and researchers will be held March 29-30 at the Arnold and Mabel Beckman Center of the National Academies of Sciences and Engineering, adjacent to the University of California at Irvine.

More than 60 researchers and scientists from North America and Europe are expected to attend the symposium, which is hosted by the Cystinosis Research Foundation, Irvine, Calif. The goal of the symposium is to promote collaborations among researchers seeking better treatments and a cure for cystinosis, the rare and fatal disease that afflicts an estimated 500 people, mostly children, in the United States and 2,000 worldwide.

"This represents the largest gathering of scientists in this rare-disease field. Even though most of these researchers have been conducting bench and clinical studies on cystinosis for several years, this is a unique opportunity for them to meet and discuss their research. It fosters collegiality and research collaboration to advance treatments and the search for a cure," said Nancy Stack, CRF Co-Founder and Trustee.

Cystinosis is a metabolic disease that slowly destroys every organ in the body, including the liver, kidneys, eyes, muscles, thyroid and brain. In patients with cystinosis, the amino acid cystine accumulates in the tissue due to the inability of the body to transport cystine out of the cell. This causes development of crystals, resulting in early cell death. There is a drug that can prolong the patient's life, but there is no cure. Almost all sufferers succumb before 40 years old and average life expectancy is 28.5 years.

In the last 10 years the CRF has funded 90 studies and fellowships with more than $17.5 million. The CRF is the leading private fund provider of grants for cystinosis research in the world.

Keynote addresses will open the two-day event. Cystinosis researcher emeritus Jerry Schneider, M.D., is honorary chair. Co-chairs are pre-eminent researchers Corinne Antignac, M.D., Ph.D., Stephanie Cherqui, Ph.D., Julie Ingelfinger, M.D., Ph.D., and Elena Levtchenko, M.D., Ph.D.

Raptor Pharmaceutical Corp. of Novato, Calif., is a sponsor of this and previous Symposiums, which were held in 2008 and 2010.

The Cystinosis Research Foundation is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to finding a cure for this devastating disease. The CRF raises funds to support bench and clinical research toward improved treatments and a cure for cystinosis. The CRF also seeks to educate the medical and public communities about cystinosis to ensure early diagnosis and proper treatment.

For more information, call the Cystinosis Research Foundation at 949-223-7610 or visit

Contact Information

  • Contact:
    Zoe Solsby, CRF
    (949) 223-7610

    Art Barrett
    (714) 602-6021