busulfan,exagamglogene autotemcel
otsuka,CRISPR,Vertex
['busulfan']
exa-cel 42/44 with b-thal stopped transfusion, with scd were free of acute voe-1 death from covid-19
72/75 with excellent outcomes-requires high dose busulfan prep #ash22 #curescd
Adult and pediatric hematologist, sickle cell provider and advocate