Each year last day of February is marked as the Rare Disease Day. It is a great and a very meaningful opportunity to raise awareness about conditions that are rare i.e. affect fewer than 1 in 1,500 people in the United States or fewer than 1 in 2,000 people in the EU. Nevertheless, they are no less important.
GCT has contributed to the development of a number medical products for rare diseases and orphan drugs in hematology, dermatology, rare CNS indications, genetic disorders, respiratory, metabolic, cancer etc. Currently, we are conducting a pediatric study in patients with a rare neurological disorder. All of us at GCT look forward to seeing the positive impact of the drug on the young patients that participate in the trial and on their families.