Rare Diseases and Orphan Drugs

Clinical case studies on Rare Diseases

The clinical presentation, explanation, pathophysiology, and sometimes mysterious nature of rare diseases have fascinated physicians for hundreds of years. Rare diseases offer opportunities to review human physiology and bioscience from distinctive views. Major scientific breakthroughs ensuing from investigation of rare diseases have usually provided insight into a lot of common disorders. The satisfaction of diagnosis a patient with a rare disorder with success is commonly chop-chop countered by the conclusion that the flexibility to know and treat the patient’s condition is restricted by mental object and also the difficulties of finding out the malady. Clinical investigators in rare malady analysis additionally frequently face challenges specific to the study of uncommon disorders. Maybe the foremost frequent downside is that the enlisting of a requisite variety of study subjects for Associate in Nursing data-based cohort or a run. This would like needs the creation of multi-institutional and international collaborations to conduct clinical investigation in rare diseases