Despite rapid therapeutic advances in sickle cell disease, from red blood cell exchange to gene therapies, delivery remains constrained by infrastructure, geography, and care fragmentation.
Sickle cell disease (SCD) is the world’s most common monogenic disorder, affecting millions globally. Yet despite its prevalence, it has endured decades of underinvestment and limited therapeutic development, resulting in stark and persistent inequities in care.
SCD is a multi-organ condition that can lead to stroke, pulmonary hypertension, kidney failure, vision loss, avascular necrosis, and chronic disability. In high-income countries, comprehensive care programs have dramatically improved survival, with most affected infants now reaching adulthood. But living longer does not necessarily mean living well. Therapeutic options remain limited, and existing treatments are significantly underused, with 70-75 percent of patients not receiving any disease-modifying treatments.