Haemoglobinopathies

Sickle Cell Disease: The Drugs That Change the Course

A single letter is wrong in the DNA, and a child grows up counting years in hospital admissions. For decades all we offered was pain relief and transfusions. Then came a daily capsule that switches a silenced fetal gene back on, an antibody that unglues cells from vessel walls, and a molecule that keeps haemoglobin in the shape that never sickles. This is the story of how sickle cell disease went from a life managed to a disease modified.

Thalassaemia and Iron Chelation: The Price of Regular Transfusion

A transfusion keeps a child with severe thalassaemia alive — but every bag of blood smuggles in iron the body has no way to throw out. Kept up month after month, that iron silently loads the heart until it fails. The story of thalassaemia treatment is really two battles: replacing the blood, then racing to clear the iron the blood leaves behind. Win only the first and the patient still dies — of the cure.