Removal of galactose binding domains from #AAV prevented complement activation in NHP. Regardless of antibodies. We have a lot to learn about AAV-associated TMA. #ASGCT Andrew Baik, Regeneron

Nice work Alexis Thompson Children's Hospital

A definitive genetic diagnosis can be transformative in #raredisease. It can provide access to life changing treatments (like in this case), prognostic information and guidance for screening, and introduction to a communities with similar problems. nytimes.com/2024/06/05/mag…

Stacey Croteau presented the Spark Phase 2 hemophilia A gene therapy. Stable FVIII but we need more patients and longer follow up. Phase 3 study started. #ISTH2024

I suspect Brian O Mahony understands more about how decisions about paying for #hemophilia drugs are made in different countries than almost anyone. 🧵👇

Another example why #pediatrics #hematology is so interesting. A normal ALC for infants is 3,500-13,000. An ALC < 2,000 in an infant is VERY abnormal and would consider SCID until proven otherwise.

Great episodes (as usual) on #pediatric rabies. A VERY helpful video about how to safely "Catch the Bat!" is at: health.ny.gov/diseases/commu… For more info about bat exposed toddlers: x.com/DrSamelsonJone…

Pls check out our work suggesting that #emicizumab (and other #FVIII-mimetics, more to come soon 😜) can likely be repurposed for select people with #hemophilia B. ashpublications.org/blood/article/… With the best collaborators: Bhavya Doshi Lindsey A. George MD Allyson Pishko, MD MSCE

So, yesterday was wild. Started off with this text from my neighbor. He's a great guy but has no business cutting open walls by himself. I grabbed some tools and went over there. 1/x 🧵

This study reports an enhanced-function #FVIII variant (R336Q/R562Q) for durable #hemophiliaA gene therapy in male mice. They demonstrate that the loss of FVIII levels is due to declines in vector copy number ⁦Lindsey A. George MD⁩ ⁦Nature Communications⁩ nature.com/articles/s4146…

Pneumococcal vaccine recommendations are getting complicated, doubly so for people with immunosuppression. I just learned about this very helpful app: cdc.gov/pneumococcal/h… #hematology #Pediatrics

Register for a Sept. 20 in-person event at Children's Hospital, The Safe and Effective Practice of AAV #GeneTherapy! During this full-day seminar, you'll hear from ASGCT Board Member Lindsey A. George MD, Past President Beverly Davidson, Denise Sabatino, + more. bit.ly/4g35xkG

W/ Bhavya Doshi, Susan Matesanz

The kids are all right 🧵👇

Last week to register for our symposium on the clinical practice of #AAV based #genetherapy. w/ Lindsey A. George MD Denise Sabatino Bhavya Doshi Susan Matesanz

Cheers to STAT and Usha Lee McFarling for writing a brilliant piece on a nuanced subject that impacts millions of people. Read about our Duffy work and impact on patients here 👉statnews.com/2024/09/09/emb… Brigham and Women’s Hematology Dana-Farber News Mass General Cancer Center Mass General Brigham Brigham and Women’s D & I

Out in #Blood this week, our work showing emi could be repurposed for some #hemophilia B. W/ Bhavya Doshi Lindsey A. George MD Allyson Pishko, MD MSCE Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab ashpublications.org/blood/article/…