How many people live with a given rare disease? How many more remain undiagnosed? Can we develop a formula to predict improvement in diagnosis rates across diseases? A recent article by the International Society for Pharmacoeconomics and Outcomes Research attempts to answer these riddles.
On May 19, the Wall Street Journal revisited industry and patient relationships leading up to FDA approval of Sarepta’s eteplirsen. Chris Smith weighs in.
As hundreds of gene therapies advance through clinical trials, a genomic revolution is taking place before our eyes. Are we ready?