Pharmaceutical Sciences

Advancing Research for rare diseases

Rare disease research is difficult due to the scarcity of patient information on the conditions. Because there are so few people affected, patient recruitment for clinical studies might be very difficult. To address these difficulties, a number of charities, patient advocacy organizations, and government agencies have developed online registries to collect patient data for researchers, doctors, and public health experts to use. It's crucial to remember that study into the aetiology, processes, and treatment approaches of rare diseases can help to advance advancements in common diseases. Rare diseases are biologically complex, and scientists are working hard to figure out what causes them and how they progress clinically. As researchers identify the molecular and genetic drivers of many diseases, we've witnessed amazing progress in the creation of medicines to treat patients with rare diseases.