Addressing challenges in the diagnosis and treatment of rare genetic diseases

Nature Reviews Drug Discovery 17, 151 (2018). doi:10.1038/nrd.2017.246 Authors: Kym M. Boycott & Diego Ardigó The past 5 years have seen an unprecedented rate of discovery of genes that cause rare diseases and with it a commensurate increase in the number of diagnosable but nevertheless untreatable disorders. Here, we discuss the increasing opportunity for diagnosis and therapy of rare diseases
Source: Nature Reviews Drug Discovery - Category: Drugs & Pharmacology Authors: Tags: Comment Source Type: research