For how we live.
A modelling analysis puts 2.75 million children under five in sub-Saharan Africa living with sickle cell disease. Seven African countries have transplant programmes; approved gene therapies cost millions.
27 of 28 patients had no severe pain crises after treatment with reni-cel. But the sponsor ended the study early, and the reported analysis was not the one it was designed to test.
Two trials showed more deaths on voxelotor than placebo. The registry data that triggered the 2024 precautionary suspension did not confirm the original signal — the trials did something worse.