ISTH Day 1

Hemophilia is a hot topic: on one hand after long half-life products are available optimal replacement therapy can be redefined balancing higher trough levels (i.e. improving bleeding prevention), interval between infusions and total dose infused/cost. On the other hand non-replacement therapy is in advanced development stage (emicizumab, concizumab, fitusiran, etc.), and gene therapy continues making steady progress.

Look for the State of the Art session tomorrow morning on novel therapies for hemophilia!