Sanofi and its development partner, the Swedish drug company Sobi, said Wednesday they plan to submit an experimental, longer-lasting hemophilia treatment for approval now that …

Gene therapies have the potential to essentially fix many diseases. Yet, those currently in development are administered just once, raising questions about whether their effects …

In both hemophilia and Duchenne, unexpected findings have led Pfizer to redraw its development plans. The company recently paused patient screening and dosing in a …