Tag

sickle cell disease

Gene-editing treatment Casgevy extended to UK children aged five

The MHRA has extended Casgevy to children from age five with severe sickle cell disease or thalassaemia, and NICE has widened NHS managed access to match.

30 October 2026

Illustration comparing normal round red blood cells flowing freely with sickle-shaped cells blocking a blood vessel

Rare Diseases

Sickle cell tablet lifts blood counts but pain crises barely move

In the Lancet-published RISE UP trial, mitapivat raised haemoglobin in 41% of people with sickle cell disease against 3% on placebo, but pain crises didn't fall significantly.

22 October 2026