Ben Samelson-Jones, MD, PhD
@DrSamelsonJones
Pediatric Hematologist @childrensphila | Gene Therapist | Husband
| 👩👦👧👶 🐕 Dad | #WilliamsSyndrome advocate | Views my own
ID:1189278099097247747
https://www.research.chop.edu/people/benjamin-j-samelson-jones 29-10-2019 20:30:01
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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:
twitter.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.
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
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…
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
Did you know that if your dog has #hemophilia they can be treated with gene therapy? This study reports the long term results of 12 companion dogs with hemophilia treated with gene therapy. Reduction in bleed rate and improvement in quality of life. tinyurl.com/fuxtp3hp
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🤔Why is pulmonary embolism (PE) relatively rare in those with Factor V Leiden?
This Factor V Leiden Paradox was pointed out to me by Ben Samelson-Jones, MD, PhD after I posted about a similar difference with Behçet Syndrome.
Let's have a look.
