Update: 27 September 2019

Rapamycin, a drug widely used for almost 20 years to protect organ transplant patients, has been found to reduce, in mice, the build up of toxic proteins that destroy red blood cells in β-thalassaemia.

Investigators are moving forward to design a small proof-of-concept clinical trial to test the safety and efficacy of rapamycin for treatment of β-thalassemia.