Ben Samelson-Jones, MD, PhD
@samelsonjones.bsky.social
Pediatric Hematologist 🧪🩸| Gene Therapist | Biochemist | 👩🦱👨👩👦🐕 Dad | Husband | Williams Syndrome Advocate
#Medsky #Hemesky
#Medsky #Hemesky
June 25, 2025 at 2:42 AM
June 24, 2025 at 9:28 PM
June 22, 2025 at 9:45 PM
Potential emerging signal difference in ADAs in next-generation FVIII(a)-mimetics. 0/61 for Mim8 but 2/30 for NXT007. Surveillance may not be the same.
#ISTH2025 #hemophilia #hemesky
#ISTH2025 #hemophilia #hemesky
June 22, 2025 at 8:42 PM
Potential emerging signal difference in ADAs in next-generation FVIII(a)-mimetics. 0/61 for Mim8 but 2/30 for NXT007. Surveillance may not be the same.
#ISTH2025 #hemophilia #hemesky
#ISTH2025 #hemophilia #hemesky
#Hemophilia A patient treated with Hympavzi developed spontaneous DVT 3 years into treatment.
Factor V Leiden heterozygous and sedentary lifestyle identified as risk factor.
#ISTH2025
Factor V Leiden heterozygous and sedentary lifestyle identified as risk factor.
#ISTH2025
June 22, 2025 at 7:32 PM
#Hemophilia A patient treated with Hympavzi developed spontaneous DVT 3 years into treatment.
Factor V Leiden heterozygous and sedentary lifestyle identified as risk factor.
#ISTH2025
Factor V Leiden heterozygous and sedentary lifestyle identified as risk factor.
#ISTH2025
Though pediatric #thrombosis is also distinct from adults
June 22, 2025 at 12:50 PM
Though pediatric #thrombosis is also distinct from adults
Pediatric #thrombosis guidelines, guidance, and clinical trials need to distinguish between older and younger children.
#ISTH2025 #hemesky
#ISTH2025 #hemesky
June 22, 2025 at 12:28 PM
Pediatric #thrombosis guidelines, guidance, and clinical trials need to distinguish between older and younger children.
#ISTH2025 #hemesky
#ISTH2025 #hemesky
Sabatino from @chopresearch.bsky.social giving a comprehensive overview of genotoxicity after AAV #genetherapy.
June 21, 2025 at 8:01 PM
Sabatino from @chopresearch.bsky.social giving a comprehensive overview of genotoxicity after AAV #genetherapy.
Rate of immunosuppression after #genetherapy for #hemophilia B with #Hemgenix in the real-world seems higher than the ~20% rate reported in clinical trial. Though small numbers here, also consistent with anecdotal results.
We will need more rigorous real-world data.
#ISTH2025 #hemesky
We will need more rigorous real-world data.
#ISTH2025 #hemesky
June 21, 2025 at 6:59 PM
Rate of immunosuppression after #genetherapy for #hemophilia B with #Hemgenix in the real-world seems higher than the ~20% rate reported in clinical trial. Though small numbers here, also consistent with anecdotal results.
We will need more rigorous real-world data.
#ISTH2025 #hemesky
We will need more rigorous real-world data.
#ISTH2025 #hemesky
Nuanced points about the use the term “Cure” when discussing #genetherapy for #hemophilia at #ISTH2025.
I agree about avoiding “cure” in clinical setting but really liked that this might be different in discussions about Allocations or Research Funding.
I agree about avoiding “cure” in clinical setting but really liked that this might be different in discussions about Allocations or Research Funding.
June 21, 2025 at 4:26 PM
Nuanced points about the use the term “Cure” when discussing #genetherapy for #hemophilia at #ISTH2025.
I agree about avoiding “cure” in clinical setting but really liked that this might be different in discussions about Allocations or Research Funding.
I agree about avoiding “cure” in clinical setting but really liked that this might be different in discussions about Allocations or Research Funding.
Can't wait for #ISTH2025 to start! Our group from
@chopresearch.bsky.social is presenting the first abstract and the last, and a few in between, all about #hemophilia B.
#Hemesky
@chopresearch.bsky.social is presenting the first abstract and the last, and a few in between, all about #hemophilia B.
#Hemesky
June 21, 2025 at 11:57 AM
Can't wait for #ISTH2025 to start! Our group from
@chopresearch.bsky.social is presenting the first abstract and the last, and a few in between, all about #hemophilia B.
#Hemesky
@chopresearch.bsky.social is presenting the first abstract and the last, and a few in between, all about #hemophilia B.
#Hemesky
I highly recommend any of Barton Roueche’s collections to understand what Epidemic Intelligence Service officers do.
Who else are you going to call when 11 blue men show up in your ER?
#medsky
Who else are you going to call when 11 blue men show up in your ER?
#medsky
February 15, 2025 at 3:49 PM
I highly recommend any of Barton Roueche’s collections to understand what Epidemic Intelligence Service officers do.
Who else are you going to call when 11 blue men show up in your ER?
#medsky
Who else are you going to call when 11 blue men show up in your ER?
#medsky
The whole quote is also worth highlighting:
February 2, 2025 at 11:48 PM
The whole quote is also worth highlighting:
#hemesky
Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for #Hemophilia A presented at #ash and published in @nejm.org
www.nejm.org/doi/full/10....
Aggressive FVIII replacement with EHL-FVIII during thrombocytopenia.
Fludarabine included to ⬇️ risk of inhibitor with FVIII-ET3
Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for #Hemophilia A presented at #ash and published in @nejm.org
www.nejm.org/doi/full/10....
Aggressive FVIII replacement with EHL-FVIII during thrombocytopenia.
Fludarabine included to ⬇️ risk of inhibitor with FVIII-ET3
December 10, 2024 at 3:42 AM
#hemesky
Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for #Hemophilia A presented at #ash and published in @nejm.org
www.nejm.org/doi/full/10....
Aggressive FVIII replacement with EHL-FVIII during thrombocytopenia.
Fludarabine included to ⬇️ risk of inhibitor with FVIII-ET3
Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for #Hemophilia A presented at #ash and published in @nejm.org
www.nejm.org/doi/full/10....
Aggressive FVIII replacement with EHL-FVIII during thrombocytopenia.
Fludarabine included to ⬇️ risk of inhibitor with FVIII-ET3
This is a long but absolutely horrific read. Melodramatic as a TV show. Touches on the nature and abuse of power in health care systems.
www.propublica.org/article/thom...
www.propublica.org/article/thom...
December 9, 2024 at 5:17 PM
This is a long but absolutely horrific read. Melodramatic as a TV show. Touches on the nature and abuse of power in health care systems.
www.propublica.org/article/thom...
www.propublica.org/article/thom...
New structural data of FVIIIa/FIXa complex at #ASH24!
FIX-R338 poised to interact with FVIIIa.
Consistent with our previous conclusion that the hyperactivity of FIX-R338L (Padua) is due improved interactions with FVIIIa.
Paper No: 428, Kenneth C Childers
insight.jci.org/articles/vie...
FIX-R338 poised to interact with FVIIIa.
Consistent with our previous conclusion that the hyperactivity of FIX-R338L (Padua) is due improved interactions with FVIIIa.
Paper No: 428, Kenneth C Childers
insight.jci.org/articles/vie...
December 9, 2024 at 4:30 PM
New structural data of FVIIIa/FIXa complex at #ASH24!
FIX-R338 poised to interact with FVIIIa.
Consistent with our previous conclusion that the hyperactivity of FIX-R338L (Padua) is due improved interactions with FVIIIa.
Paper No: 428, Kenneth C Childers
insight.jci.org/articles/vie...
FIX-R338 poised to interact with FVIIIa.
Consistent with our previous conclusion that the hyperactivity of FIX-R338L (Padua) is due improved interactions with FVIIIa.
Paper No: 428, Kenneth C Childers
insight.jci.org/articles/vie...
Novel FXa-DOAC bypassing agent VMX entering Phase 3. Timely, given recent thrombotic concerns for Adexanet, see shows.acast.com/dont-just-re... (Episodes 5, 6).
I am curious about anti-drug antibodies.
@mettinebos.bsky.social
@richardbuka.bsky.social
#Ash24 #hemesky
I am curious about anti-drug antibodies.
@mettinebos.bsky.social
@richardbuka.bsky.social
#Ash24 #hemesky
December 9, 2024 at 3:58 AM
Novel FXa-DOAC bypassing agent VMX entering Phase 3. Timely, given recent thrombotic concerns for Adexanet, see shows.acast.com/dont-just-re... (Episodes 5, 6).
I am curious about anti-drug antibodies.
@mettinebos.bsky.social
@richardbuka.bsky.social
#Ash24 #hemesky
I am curious about anti-drug antibodies.
@mettinebos.bsky.social
@richardbuka.bsky.social
#Ash24 #hemesky
Enhancing membrane FIXa binding with novel FVIII-mimetics has substantial procogulant effect. Opens new avenues for designing #hemophilia therapies.
Paper No: 427
Shekhar Kumar
#ASH24 #Hemesky
Paper No: 427
Shekhar Kumar
#ASH24 #Hemesky
December 8, 2024 at 6:39 PM
Enhancing membrane FIXa binding with novel FVIII-mimetics has substantial procogulant effect. Opens new avenues for designing #hemophilia therapies.
Paper No: 427
Shekhar Kumar
#ASH24 #Hemesky
Paper No: 427
Shekhar Kumar
#ASH24 #Hemesky
We are presenting preclinical and clinical gene therapy studies for #hemophilia tonight at #ASH24 poster session. Lots to discuss.
December 8, 2024 at 3:58 PM
We are presenting preclinical and clinical gene therapy studies for #hemophilia tonight at #ASH24 poster session. Lots to discuss.
And C1-inhibitor haploinsuffiency increases venous #thrombosis.
December 8, 2024 at 1:13 AM
And C1-inhibitor haploinsuffiency increases venous #thrombosis.
#Ash24 #hemesky
Very large datasets suggest factor 12 haploinsufficiency substantially reduce venous #thrombosis.
Suggest intravascular pathological clot formation is molecularly different than extra vascular hemostatic clot formation?
Very large datasets suggest factor 12 haploinsufficiency substantially reduce venous #thrombosis.
Suggest intravascular pathological clot formation is molecularly different than extra vascular hemostatic clot formation?
December 7, 2024 at 9:31 PM
#Ash24 #hemesky
Very large datasets suggest factor 12 haploinsufficiency substantially reduce venous #thrombosis.
Suggest intravascular pathological clot formation is molecularly different than extra vascular hemostatic clot formation?
Very large datasets suggest factor 12 haploinsufficiency substantially reduce venous #thrombosis.
Suggest intravascular pathological clot formation is molecularly different than extra vascular hemostatic clot formation?