Nice work Alexis Thompson Children's Hospital

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

Come see CHOP Research at #ASGCT2024

1658 Evolution Directed Gene Engineering (EDGE) Identifies Factor VIII Variants Augmented for #GeneTherapy in #Hemophilia A Mice

1834 A Centralized Multi-Disciplinary Approach for Safe and Efficient Delivery of In VivoGene Therapy

It’s Williams syndrome awareness month. Our daughter has Williams syndrome. Neuroscientists are confounded due to the developmental delays and cognitive challenges coming with high verbal abilities, highly social personalities, and an affinity for music. #WilliamsSyndrome

This is medicine by press release, as I discussed in Clinical Trial Considerations in AAV Gene Therapy this morning at #asgct

#SMA occurs in ~1 of 10,000 babies. Nobody plans for a baby having a rare genetic disorder. I know because this happened to our family. This is the rationale for medical insurance: to spread risk across many.

Mosaic Life Care: your inadequacy should not be a death sentence.🤬

Before #GeneTherapy , SMA was the most common monogenic cause of infant mortality.

#ASGCT Susan Matesanz

Insurance Denies Newborn Twins Life-Saving Treatment for Fatal Genetic Disorder: 'Hard to Cope' people.com/insurance-deni… via People

Agree with everything except done at CHOP Research

The FDA approval of #Beqvez is another milestone in #hemophilia and #genetherapy .

Like #Hemgenix , this #AAV vector uses the hyperactive FIX-Padua.

I wrote about the widespread adoption of FIX-Padua:
cell.com/molecular-ther…

Also, detailed tweetorial:
x.com/drsamelsonjone…

It is a great day because Maryland’s stock albuterol law will be signed by Gov. Wes Moore today. It was a five year labor of love, but we did it! I will miss the bill signing because I am seeing patients in clinic, but feeling very thankful today for all who supported this effort.

🩸 Peds heme/onc fellows and early faculty 🩸

On #WorldHemophiliaDay , meet the Ward family. Melody’s sons, Jadon and Roan, were born with hemophilia, an inherited bleeding disorder that prevents blood from clotting properly. The disease runs in Melody's family, going back at least 5 generations. ms.spr.ly/6016Y6Aji

Happy #WorldHaemophiliaDay

Marked on 17th April, which was the birthday of Frank Schnabel, founder of the WFH

Off into London for a board meeting of Haemophilia Society

Much to discuss!

#Haemophilia

#Hemophilia

#BleedingDisorders

#Green plasma! Great 🧵👇. Happy ☘️

#MedTwitter
#hematology

Factor levels in #hemophilia from biochemistry to clinical considerations.

Such a fun conversation.

W/ Patrick James Lynch Radoslaw Kaczmarek Peter Lenting Courtney Thornburg.

Register for THSNA 2024 today! Registration pricing increases tomorrow, March 5! #THSNA2024 thsna.org/2024/attend-re…

No lead level is safe for children. 😡😡😡 nytimes.com/2024/02/27/hea…

Have your social media pledge cards ready? Downloadable in 8 languages from our website, show your support for Rare Disease Day now! Get them here: rarediseaseday.org/downloads/ #RareDiseaseDay #ShareYourColours #Awareness #LightUpForRare