Track: Medical plenary
Maria Elisa Mancuso, MD, PhD
Hematologist
IRCCS Humanitas Research Hospital
Milan, Italy
The advent of both FVIII/FIX molecules with enhanced pharmacokinetic properties and non-replacement hemostatic agents has raised the bar of protection from bleeds in hemophilia. This has shifted the paradigm towards the transformation of severe phenotype into mild in order to additionally achieve long-term joint health outcomes. Moreover, the transition to mild phenotype can be considered as an acceptable trade-off for current non-curative gene therapy approaches.
Speaker: Maria Elisa Mancuso, MD, PhD – IRCCS Humanitas Research Hospital