Cytonet Recognizes Rare Disease Day with Efforts to Help Ensure UCD Diagnoses Are Not Missed
HEIDELBERG, Germany, Feb. 27, 2012 /PRNewswire/ -- For parents of babies born with urea cycle disorders (UCD), the joy of giving birth to a perfect baby can quickly turn to devastation as the baby's health rapidly declines, going from symptoms such as being tired and irritable, to not eating, to experiencing seizures and coma. UCDs are congenital and often life-threatening disorders of ammonia metabolism in the liver that cause neurotoxic ammonia to accumulate in the body, potentially leading to massive damage of the nerves and the brain and death.
UCDs are one of more than 7,000 rare diseases. In recognition of Rare Disease Day on February 29, international biotechnology firm Cytonet is taking several actions to help make a difference for people affected by UCD. "Cytonet is deeply committed to improving the lives of people with UCD," says Dr. Wolfgang Rudinger, CEO and CSO of Cytonet Germany. "It is critical that symptoms are recognized and a diagnosis of UCD is made as quickly as possible to give these children the best chance, so we are helping educate healthcare professionals and the public about UCD." Activities include:
- Cytonet sponsored the newly released‚ "Physician's Guide to Urea Cycle Disorders," published by the National Organization for Rare Disorders (NORD) to educate healthcare professionals about symptoms, making the diagnosis and treatment.
- Cytonet produced a short video for medical professionals about UCD and a clinical trial for liver cell therapy, which is currently enrolling patients.
- Cytonet is exhibiting at Rare Disease Day at NIH on Feb. 29, a day-long celebration and recognition of the various rare diseases research activities supported by the NIH Office of Rare Diseases Research, the NIH Clinical Center, other NIH Institutes and Centers; the Food and Drug Administration's Office of Orphan Product Development; the National Organization for Rare Disorders; and the Genetic Alliance.
- Cytonet is sponsoring an exhibit at NEO, the conference for Neonatology, this week and providing attendees with information on recognizing the symptoms and diagnosing UCD.
- And, Cytonet is collaborating with the National Urea Cycle Disorders Foundation (NUCDF) to help them create disease awareness programs which will launch later this year.
Children who remain untreated for UCD rarely experience normal physical and mental development. The only cure is liver transplantation, which can be an extremely difficult procedure for very young children and neonatal patients. Additionally, there is a shortage of suitable organs available for transplantation.
Cytonet's SELICA III clinical trial is designed to test the safety and efficacy of liver cell therapy in infants to children up to five years old with UCD. The trial will enroll 20 participants nationally. Liver cell therapy involves collecting healthy cells from donated livers not suitable for transplantation (obtained from U.S. organ procurement organizations) which are then gently isolated and undergo complex processing. These cells are infused into the portal vein over six days. SELICA III is currently enrolling patients at 14 sites in the United States and Canada. For more information, please visit Cytonetllc.com. For more information on UCD, please visit nucdf.org.
About Cytonet
Cytonet is an internationally active biotechnology company which is located in Weinheim and Heidelberg in Germany and in Durham, NC in the U.S. The company develops and produces cell therapeutic products. Cytonet's goal is to provide alternatives to existing therapies for many diseases with a particular emphasis on liver diseases. Cytonet is a pioneer and leader in the field of regenerative medicine. For the past several years, Cytonet has worked with internationally-leading metabolism and neonatal centers to study its liver cell therapy, which uses healthy and metabolically functional human liver cells collected from donated livers not suitable for transplant for infusion to treat urea cycle disorders in children. For more information, please visit Cytonet's website at Cytonetllc.com.
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SOURCE Cytonet
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