The Priority Review

Tag

sickle cell disease

1 story

  1. Clinical trials

    Mitapivat shows haemoglobin response in sickle cell disease trial

    In the RISE UP phase 3 trial, 56 (41%) of 138 patients on mitapivat achieved a haemoglobin response versus 2 (3%) of 69 on placebo, an adjusted difference of 38% (95% CI 29-47). The annualised rate of pain crises was 2·6 for mitapivat and 3·1 for placebo (p=0·1213).