Pfizer and Sangamo have restarted recruitment of patients into their registration trial for haemophilia A gene therapy giroctocogene fitelparvovec, after the FDA lifted a clinical hold on t
BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe
Novo Nordisk has reported phase 3 results with its concizumab drug for haemophilia A or B, showing efficacy in the prevention of bleeding events that will support regulatory filings startin
Sanofi R&D in its core haemophilia category is advancing on three fronts – engineered proteins, RNA interference drugs and gene therapies – and highlighted results in two of those at th
