Rare diseases can be described in terms of incidence, etiology, morbidity, and survival. Incidence can vary, however, from rare (e.g., Gaucher disease, cystic fibrosis) to epidemic proportions (e.g., HIV, malaria, cholera). The incidence of less frequently occurring...
In general, drugs often work in only half of the people who take them. In addition, billions are spent to treat adverse drug reactions and other complications. The personalized medicine approach is to elucidate the differences between patients who respond to a drug...
As the old “blockbuster” mentality fades in the wake of relatively weak pipelines, increased emphasis on drug safety, slow sales growth, and the loss of patent protection on older blockbusters, it would appear that the orphan-disease niche (i.e., low...
