A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers ...

Liver disease includes any condition that damages your liver. Over time, scar tissue can build up in your liver. Scarring or fibrosis can progress over many years until you develop cirrhosis—the most ...

Rare diseases affect less than 5 people out of 10,000. However, this still amounts to about 7% of the world’s population, with over 10,000 such conditions. Almost all are genetic in origin, with a few ...