SOURCE: Cure Cystinosis International Registry
IRVINE, CA--(Marketwire - Jan 5, 2012) - More than 300 people from 31 countries have registered in the Cure Cystinosis International Registry, the first global effort to link cystinosis patients with scientists and researchers who are developing new treatments and conducting clinical trials. The rare metabolic and disorder affects about 2,000 people, mostly children, worldwide.
The CCIR also is launching a new feature of the registry, Frequently Asked Questions, a section that contains answers from researchers and scientists to inquiries pertaining to the disease and its treatment.
"I have no doubt that this new forum will stimulate research on cystinosis in the future and I encourage every cystinosis family to join," said Dr. Paul Goodyer of the Montreal Children's Hospital, a cystinosis researcher who also helped with the site's language translations.
The CCIR is the only international patient registry dedicated to the cystinosis community and represents a collaborative effort of 14 cystinosis advocate foundations worldwide, said Nancy Stack, President and co-founder of the Cystinosis Research Foundation.
"Top cystinosis researchers are encouraging cystinosis patients to register with CCIR. Our goal is to have every cystinosis patient worldwide register, which is made easier because the site offers translations in Spanish, French and Portuguese," Stack said. "
The purpose of the CCIR is to identify people with cystinosis globally and collect their medical histories and information to share (anonymously) with the cystinosis research community. This information will allow clinicians, researchers and pharmaceutical companies to accelerate novel treatments and a cure for cystinosis.
Researchers Urge CCIR Enrollment
The importance of the CCIR is recognized by clinicians and researchers whose work on cystinosis and with patients is advanced by increased participation of the cystinosis community.
"The registry allows us to focus for almost the first time on directions that are particularly relevant to the patients themselves," said Dr. Ranjan Dohil of the University of California, San Diego.
Patrick Harrison, Ph.D. and head of the physiology department at University College Cork, Ireland, said, "As a scientist, the registry is a unique and critically important resource, especially now."
Many researchers worldwide also have registered with the CCIR. Stack said this clearly demonstrates the importance of the patients' information, particularly to researchers doing advanced bench and clinical studies.
Patient Information Is Private, Protected
The CCIR contains current information regarding cystinosis clinical trials and studies. All patient information is de-identified and held in a secure data base accessible only by the CCIR curator. Participants are able to access aggregate data allowing them to view how they fit within the larger cystinosis community.
CCIR officials say recruitment for clinical trials can be a lengthy process, especially for a rare disease like cystinosis. Participation in this registry will speed up the recruitment process and facilitate and expedite clinical trials.
In patients with cystinosis, the amino acid cystine accumulates in the tissue due to the inability of the body to transport cystine out of one of the compartments of the cell. Cystinosis is a metabolic disease that slowly destroys every organ in the body, including the liver, kidneys, eyes, muscles, thyroid and brain. There is a treatment for cystinosis but there is no cure. Most cystinosis patients succumb to the disease or its complications by age 40.
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