Rare diseases have long been an orphan of drugs and treatments. These are diseases that affect a few people, mostly children, and are therefore difficult to study. But something is changing: thanks to scientific research and technological innovation that have made it a reality for some of these diseases to be able to correct the genetic defect that causes them. Mirko Pinotti, director of the department of Life Sciences and Biotechnology at the University of Ferrara, and Dirk Vander Mijnsbrugge, physician and Vice President Medical Affairs International Developed Markets of Pfizer Biopharma – Rare Disease, talk about it.
