exagamglogene autotemcel
CRISPR,Vertex
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#climb trial in #sicklecelldisease shows improvement in vaso-occlusion but only has 2 pts who are &gt;24 months post-tx. this study needs more time before exa-cel approval to ensure persistence of fetal hb especially since hb goes down in those patients &gt;15mo follow up. #ash22 
I specialize in sickle cell disease care for all ages with the heart of a pediatric hematologist. My tweets are my opinions to be shared and to support others