US biotech Sarepta's shares jumped by as much as 60% after a gene therapy produced dramatic results in three boys with the muscle wasting disease Duchenne muscular dystrophy (DMD).
UK patients are stepping up efforts to resolve the “heartbreaking” row that is denying their families access to Vertex’s groundbreaking cystic fibrosis (CF) drug Orkambi, after Sweden becam
A bumper crop of orphan drugs from the likes of Amicus, Kyowa Kirin, Eusa and Biogen dominates this year’s UK Prix Galien shortlist as biotech firms and specialist pharma companies cement t