Rare Disease Day is observed on the last day of February to raise awareness for rare diseases and improve access to treatment and medical representation for individuals with such conditions.
There are more than 8,000 rare diseases and about 8% of the global population has a rare disease. Given the fact that the prevalence of such diseases is low, drug research and development is often slow and people living with a rare condition are overlooked.
GCT is actively participating in the research and development of orphan drugs. The most recent projects we have been involved in include Dravet Syndrome, Kabuki Syndrome, Primary Mitochondrial Myopathy – rare CNS diseases, Type II Mucopolysaccharidosis – endocrinological disease, and Idiopathic Pulmonary Fibrosis – a rare pulmonary condition.
Reach out to us at bd@gctrials.com to find out more about our experience with managing Rare disease clinical trials.