#genetherapy for #hemophilia can be life changing. Proud to offer this transformative treatment at @childrensphila.bsky.social Gene therapy changed the life of a South Jersey man with hemophilia | PhillyVoice www.phillyvoice.com/gene-therapy...

I’m honored to be speaking at the 14th WFH Global Forum in Montreal, Nov 13–14, 2025! 🌍 Join us for global insights on #BleedingDisorders care—covering #GeneTherapy, #VWD, safety, and more—all through the lens of equitable access. Learn more 👉 wfh.org/global-forum/

I don’t even know where to start. Trump administration blocks NIH from awarding any research grants and contracts www.statnews.com/2025/07/29/t... via @statnews.com

As #hemophilia B therapeutics become more sophisticated, there is an ⬆️ need to better understand genotype–phenotype relationships. Our in vitro studies are consistent with recent in silica approaches: www.sciencedirect.com/science/arti...

We have the best hematologists at @chopresearch.bsky.social #ISTH2025 @hilarywhitworth.bsky.social

Impaired fibrinolysis in APS, provides mechanism for elevated rate of CTEPH in APS. #ISTH2025

Brilliant visualization of extravascular FIX and very interesting (and surprising) differences in specific activity of extravascular FIX and FX in different tissues. 🤯 #ISTH2025 #hemesky

😳 Thrilled we finally have a apixaban pediatric FDA approval to implement these changes.

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

#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

Pediatric #thrombosis guidelines, guidance, and clinical trials need to distinguish between older and younger children. #ISTH2025 #hemesky

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

Compelling evidence presented at #isth2025 by Sebastian Leyes Porello from @samelsonjones.bsky.social’s group showing mim8 can enhance activity in hemophilia B variants in vitro, above enhancement this group saw with emicizumab. Curious about in vivo data teased during the Q&A 👀 #hemesky @isth.org

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.

IMO biggest update at Guideline presentation at #ISTH2025 is this recommendation for primary #VTE prophylaxis in children receiving long-term TPN. I wish there was better GUIDANCE on intensity and type of anticoagulation for this recommendation.

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

Looking forward to #ISTH2025 and especially this session with Dom. #hemophila #genetherapy

I discuss our #asgct2025 abstract highlighting our multidisciplinary team approach to care for #genetherapy recipients. Stay tuned, lots more to come. www.cgtlive.com/view/samelso...

Nice article about ongoing work to bring #genetherapy to pet dogs with #hemophilia. Includes the importance of the research colonies in developing new therapies, both for humans and 🐶. Cute pictures! Veterinarian aims to bring hemophilia treatment to pet dogs - News news.vin.com/default.aspx...

#williams syndrome is a rare genetic disorder with a fascinating but challenging neurocognitive profile. www.bbc.com/future/artic...

For the first time in 16 years, ISTH is returning to the U.S. for an in-person Congress. As we prepare to welcome the world to Washington, D.C., we’re guided by our mission & appreciation for our community🌍 Read more @jthjournal.bsky.social: www.jthjournal.org/article/S1538-7836(25)00213-2/abstract

It is essential for #genetherapy progress for unsuccessful clinicals trials to be published. We discuss recent study showing no benefit for prophylactic steroids after #AAV for #hemophilia. authors.elsevier.com/a/1k-re5TuFx... w/ @rkaczmarekdr.bsky.social

In addition to demonstrating long-term durability of rAAV #genetherapy, here we show: 1) Hemostatic potential of FIX Padua with multiple major trauma not needing additional treatments. 2) Importance of limiting hepatotoxic exposures with binge drinking associated with irreversible FIX loss.

Thrilled to mark World #Hemophilia Day with this paper showing long-term benefits after #AAV gene therapy for #hemophiliaB prn.to/4iraiEH Paper at: www.nejm.org/doi/10.1056/...

Longest follow up of expressing hyperactive factor IX Padua after #genetherapy for #hemophilia. Mostly stable transgene-product levels (except w/ binge drinking), major traumas without additional treatment, no IP related-safety concerns.

#Pediatric Scientist Development Program grant was terminated. The federal grant that has sustained PSDP for over 35 years was abruptly ended with no opportunity for appeal. This is utterly unconscionable if you care about the health of children. amspdc-psdp.org/psdp-advocac...

ASH and 90+ Organizations Call for HHS to Immediately Restore CDC’s Division of Blood Disorders. Without it, patients with blood diseases face preventable risks. ASH is calling for its restoration. 🔗 https://bit.ly/4jt5sYv #Fight4Hematology #HemeSky

Ryan White died thirty-five years ago today. To mark the occasion and to celebrate Ryan’s remarkable life, @teenvogue.com has published this excerpt from my new book. 🗃️ www.teenvogue.com/story/ryan-w...

Alternative #genetherapy approaches for #hemophilia A have recently demonstrated exciting proof-of-concept clinical results. Here I discuss 2 ex vivo approaches: www.sciencedirect.com/science/arti... For more info on current #AAV approaches, see ashpublications.org/bloodadvance...

#HTRS2025 social media ambassador. Great follow. #hemesky

Rigorous scientific evidence rarely demonstrates a treatment results in a “miraculous and instantaneous recovery.” This is the semantics of a charlatan. #Medsky www.nytimes.com/2025/03/10/h...

Phew indeed Wordle 1,350 6/6 ⬜⬜⬜⬜⬜ ⬜⬜⬜⬜⬜ ⬜⬜🟨⬜⬜ ⬜🟩⬜⬜🟩 🟩🟩⬜⬜🟩 🟩🟩🟩🟩🟩

What are doing as society that we let children die of preventable diseases? #pediatrics apnews.com/article/meas...

I discuss the biological basis of new #hemophilia therapies. ow.ly/azsR50V3XBP

At @chopresearch.bsky.social, we care for patients up to 22 years of age and have had excellent outcomes for Paget-Schroetter syndrome (effort thrombosis) with a multidisciplinary approach. #HemeSky www.tmz.com/2025/02/20/v...

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

#NIH funding is an economic driver across the country: www.unitedformedicalresearch.org/nih-in-your-...

This is going to be a problem. 😬 grants.nih.gov/grants/guide...

The most powerful laxative for a 4 yo is snowpants.

Great strides in #genetherapy.

Well, this made my day. Wishing everyone a speedy recovery.

Tonight! Come see my poster at #ash24 and learn about factor IX, warfarin and emicizumab!

#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

#ASH24 #Hemesky Long term cure in HSC based gene therapy for ADA-SCID. 95% event free survival for >10 years! Commercialized planned through Public Benefit Corporation. Novel technology and novel commercialization. Paper No: 1051 Katelyn Masiuk

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...

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...

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