Clinical Research on Rare Diseases

Clinical researchers working on rare diseases frequently encounter difficulties unique to the study of uncommon disorders. Rare diseases can offer unique possibilities to examine human physiology and biomedical science. An estimated 25–30 million persons (8–12% of the population) are thought to have one of the roughly 7000 rare diseases that affect this community. The Orphan Drug Act was passed in 1983 with the goal of encouraging the development of medications to treat, prevent, or diagnose uncommon diseases or ailments that afflict fewer than 200,000 people. Over the past two decades, there has been a significant change in both the scientific technique and the logistics of clinical research for rare diseases. The pathogenesis of the disease is now more understood, and numerous innovative treatments have been created.

  • Orphan products clinical Trials Grants Program
  • Rexin G
  • Biomedical

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