Abstract

The history of haemophilia care has been fraught with extreme successes and epic failures. The development of plasma-derived concentrates made prophylactic treatment and home care possible, but the unintended consequences were devastating for a generation and only abated with the emergence of recombinant products. Now with the arrival of longer-acting factor concentrates and the potential offered by gene therapy, further improvements in medical and social outcomes are possible. But these new treatment approaches raise challenging ethical and moral issues that society must be prepared to confront.