PhRMA: New tools, genome insight boost rare disease drug development
This article was originally published in Scrip
Executive Summary
With the advancement of new tools and a better understanding of the human genome in hand, biopharmaceutical makers are now pursuing more than 450 products aimed at treating rare diseases and genetic disorders, the Pharmaceutical Research and Manufacturers of America (PhRMA) reported in a new analysis on 7 October, declaring the current era as a "time of great progress and hope."