News
'One million Dutch people with a rare disease’
Approximately one million Dutch people are currently living with one of the estimated 6,000 rare disorders. Rare disorders are often chronic and progressive. They largely affect children who, as a result of a rare disease, will never grow old. Thirty percent of the children with a rare disorder die before they reach the age of five.
On Friday 26 February, during the Rare Disease Day, it was announced that there is great international interest in the Dutch Database of Rare Metabolic Diseases. Project leader Gepke Visser from the University Medical Center Utrecht comments: “Researchers from all over the world are showing interest, because our database is unique."
"A database like this is vital", says project coordinator Sonja van Weely. "Because every week, five new rare disorders are described. Disorders that often affect children who as a result will never grow old. This is almost completely unexplored territory."
Van Weely: "In this database, we collect all of the information on symptoms and treatments of individual patients, to gain know-how that can later be used to develop medicines." All eight clinical genetics centers in the Netherlands are working on the web-based database, which will be further developed under the umbrella of the Top Institute Pharma in Leiden.
Van Weely: "Scientists can only begin research on a certain disease when they have a group of patients. This database gives them something to start from. That is vital. We hope that the database will give a much-needed impulse to research into the development of treatments for rare metabolic disorders."
Relevant information:
Project: Sustainable orphan drug development