February 29 is World Rare Disease Day. Rare Disease Day has been held since 2008 at the initiative of the European Organisation for Rare Diseases to raise awareness of not known or neglected diseases.
Rare (or orphan) diseases are diseases that occur in a small number of people relative to the general population. Rare diseases are found in every sphere of medicine and approximately 80% of them are genetic. Symptoms of rare diseases may be evident from birth or appear during childhood. At the same time, more than 50% of rare diseases manifest in adults. Orphan diseases are characterized by different clinical symptoms, which are different even in patients suffering from the same disease. These patients have a special lifestyle, special diet, physical activity, etc.
Due to lack of knowledge about the pathology of the disease, diagnosis is delayed, which often leads to early and irreversible complications. Today, the relevance of the problem of rare diseases has increased due to the expansion of early diagnosis of this group of diseases and the emergence of effective treatment options.
The GCT team is actively involved in clinical trials of drugs for rare diseases. Reach out to us at bd@gctrials.com to find out more information.