Recent studies highlight the benefits of hydroxyurea in Hb SC disease, showing reductions in VOCs and improvements associated with induced iron deficiency. Pyruvate kinase activators demonstrate strong efficacy in SCD and thalassemia at both clinical and biological levels. Gene therapy offers sustained and impressive benefits, though further research is needed to evaluate long-term safety and complications beyond VOC reduction.
(BELG J HEMATOL 2025;16(1):29–30)
* (only possible for healthcare professionals with RIZIV/INAMI/MATRICULE working in the field of Hematology in Belgium and Luxembourg with prescription authorisation)
** (for healthcare professionals with prescribing authorisation working outside the field of Hematology in Belgium and Luxembourg as well as those without prescribing authorisation working as healthcare professional in Hematology in Belgium and Luxembourg, plus persons without prescription authorisation working outside Belgium and Luxembourg, plus healthcare professionals with prescribing authorisation outside Belgium and Luxembourg plus non-healthcare professionals from outside or inside Belgium or Luxembourg)
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