 
                                seth rotz
@sethrotz
Pediatric Hematologist/Oncologist/BMT @Cleveland Clinic Children's; opinions my own
ID: 875730816
12-10-2012 14:25:03
1,1K Tweet
551 Followers
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        Leveraging Oncology Treatments for Vascular Anomalies my.clevelandclinic.org/podcasts/cance… via Cleveland Clinic
 
         
         
        Impressive results about Exa-Cel in TDT from Vertex Pharmaceuticals at # ASH - 54 patients - 98% (53/54) had achieved Transfusion free x12 months - the remaining patient is free of RBC Tx for at least 8 months Similar results about safety and efficacy in #SCD
 
                        
                    
                    
                    
                 
        BEACON study initial data Beam Global IN #AS24 - BASE editing in Hgb1/2 for #SCD :7 patients - less collection cycles - faster engraftment in ANC (17 days) and PLT (19 days) - one death - high level of hgb F (>60%) with improvement in total hemoglobin and less hemolysis
 
                        
                    
                    
                    
                 
         
        Fred Hutch’s Mohamed Sorror presents the latest findings on a new health assessment model called CHARM, which helps health care teams predict which patients will survive transplantation. #ASH24 fredhutch.org/en/news/center…
 
                        
                    
                    
                    
                 
         
        🚨PublicationAlert🚨 How I Treat Sickle Cell Disease with Gene Therapy, published online (authors.elsevier.com/a/1kKhU1UYwnGSK ) and in print in Blood Journal Many thanks to Alexis Thompson Julie Kanter Lakshmanan Krishnamurti Shalini Shenoy MD Brandon Triplett and A Abraham for their inputs 🙏
 
                        
                    
                    
                    
                 
         
         
                         
                         
                         
                         
                         
                         
                         
                         
                        