US drug spending hits $425 billion

and immune incompatibility can lead to rejection. With Strimvelis, the patient’s own bone marrow is removed and the cells are treated with a viral vector that inserts the ADA gene into cellular DNA. Gene-corrected cells are then re-introduced into the patient. In a trial of the one-off treatment, 11 of 12 patients (92%) hit the primary endpoint of… CONTINUE READING