For how we live.
The NIH-funded STArT trial in children and young adults was halted for futility: time to crisis resolution was 60.8 hours with arginine versus 65.8 with placebo, well within the range of chance.
Across 84,348 hospital admissions, 19.8% of children had severe anaemia; as malaria receded the burden moved to older children, and severe anaemia doubled the odds of dying.
In two industry-funded phase 3 studies, every child followed at least 16 months met the goal — but all had a grade 3 or 4 event, and one died of a conditioning-drug complication.
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.