Marilyn Manco-Johnson, a leading expert in paediatric haematology, is shaping the future of haemophilia care. Her work at the University of Colorado School of Medicine and the Mountain States Regional Hemophilia and Thrombosis Center has been pivotal in transforming treatment outcomes for children. Manco-Johnson reflects on the journey from early prophylactic treatments to today's advanced therapeutic landscape.
Key challenges remain, including joint health, treatment response variability, and access to care. However, emerging innovations like gene therapy and non-factor therapies promise to redefine long-term management, addressing unmet needs and offering new hope for young patients.