Introducing new therapies – the forgotten heroes
We often hear of the endpoints, outcomes and p values associated with introducing new therapies for haemophilia. Delegates to this week’s EAHAD virtual congress heard about the burden and the benefits that are often hidden behind the numbers. For those with haemophilia who opt for gene therapy, the reality is often different to what many …
Phoenix rising – share your stories with us
Last year saw the culmination of two major consultation exercises affecting the bleeding disorder community. In May 2020 there was the review of bleeding disorder services by the Quality Review Service, followed by the November report of the All Party Parliamentary Group Inquiry into Access to Treatment. Both put forward numerous recommendations relevant to addressing …
Catch-up time for diagnosis and management of von Willebrand disease
While haemophilia is the most well-known inherited bleeding disorder, the commonest such disorder is in fact von Willebrand disease (vWD). Despite regional guidelines in countries such as the USA and UK, there has been no global agreement and endorsement from both the scientific and patient communities to drive forward better evidence-based treatment and care for …