Targeted therapy in rare cancers--adopting the orphans.

摘要: Designation of a rare 'orphan' disease is usually conferred by prevalence one in 1,500 to 2,500 individuals. Increasingly, orphan diseases are also being defined their molecular fingerprints. Rare uniquely challenging from therapeutic standpoint; it critical modify clinical study design treatments for disorders as well the increasingly smaller subsets within frequently occurring cancers. In spite immense challenges associated with developing treatment disorder, some most groundbreaking discoveries have been made malignancies. This situation may be because limited number driver aberrations occur disorders, which can targeted agents. Here, we describe drug-class examples therapies diseases, particular emphasis on malignancies or tumour-prone nonmalignant conditions, potential strategies that adopted treat these conditions.