Clinical research challenges in rare genetic diseases in Brazil

Abstract excerpt

Rare diseases are defined as conditions with a prevalence of no more than 6.5 per 10,000 people. Although each rare disease individually affects a small number of people, collectively, the 6,000 to 8,000 rare conditions (80% of them with genetic cause) affect around 8% of the world's population. Research about the natural history and underlying pathophysiological mechanisms of rare diseases, as well as clinical...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Cystic Fibrosis Research Advances
• Share to Genomics and Rare Diseases
• Share to Lysosomal Storage Disorders Research