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Chan at #eahad2025. Efanesoctocog (Altuvio) for surgery in #hemophilia

The Leloir pathway was described over 7 decades ago, there are significant shortcomings in the current approach to Galactosemia management. This new article explores possible future therapies: 👉 doi.org/10.1002/jimd.7… GalactosemiaFDN #galactosemia

I couldn't agree more! Congrats Michael Makris!

Agreed! Damien Walter

Glycolysis drives disease-specific H3K27 acetylation and transcription in CD4+ T cells of patients with juvenile idiopathic arthritis Cell Reports cell.com/cell-reports/f…

Editorial in the NEJM about the current assault on science in the US. "As editors, we should continue to do what we think makes a difference."

Obituary of an Australian blood donor. Despite having only one lung, he donated 1173 times between 1954 and 2018 and contributed to every batch of anti-D made in Australia. Amazingly, in Australia, the maximum age for blood donation is 81 years. tinyurl.com/yme2hck8

Congratulations Krzysiek Mikołajczyk !

The first woman to be awarded a Nobel Prize, the first individual to be awarded two Nobel Prizes and still today the only individual with two Nobel Prizes in two different scientific categories: Marie Skłodowska Curie. Skłodowska Curie developed the term radioactivity,

Why some amino acid substitutions create immunogenic blood group antigens (e.g. Kell), while others do not? The most immunogenic antigens are typically buried in rigid, ordered protein regions with lower mean relative solvent accessibility. tinyurl.com/mpbutbwc #blood_groups

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-re5TuFxMh… w/ Radoslaw Kaczmarek

The timely reporting of clinical trial results answers 1 unknown for hemophilia #gene therapy: no benefit of prophylactic #immunosuppression jthjournal.org/article/S1538-…

🩸Outdated therapies like cryoprecipitate pose safety risks for people with #BleedingDisorders. The WFH has submitted proposals to update 2025 WHO EML requesting to align the EML to international clinical guidelines for hemophilia and VWD. 📖Read more: bit.ly/4jxzJ8R

Thank you Michael Makris for your clear and concise commentary on our #PITT case in British Journal of Haematology. Take home message: test anyone with the combination of thrombosis and thrombocytopenia for anti-PF4 antibodies (by ELISA) and have a low threshold for starting non-heparin anticoagulation

Have you ever wondered why some glycosyltransferases recognize glycoproteins, some glycolipids, and some both? Together with Krzysiek Mikołajczyk and Radoslaw Kaczmarek, we try to answer that complicated question. #glycotime tinyurl.com/5b6kxhp3

Gene therapy for hemophilia – From basic science to first approvals of “one-and-done” therapies hubs.li/Q03mqMJl0 Special issue highlight: Celebrating 25 years of Molecular Therapy Molecular Therapy @asgctherapy Radoslaw Kaczmarek

N-glycosylation of CD4+ T cells in Graves' Disease: complex/high-Man differences between CD4+CD25− and CD4+CD25- cells influenced by MGAT5 and FUT8 expression. Pawel Link-Lenczowski tinyurl.com/y4zzx4ye #glycotime

🧬A new review explores the science behind #GeneTherapy for #hemophilia A & B—from early breakthroughs to approved AAV-based treatments. It also dives into key challenges like immune response & durability. Read the article: bit.ly/45QCRbQ

Kaczmarek #ISTH2025 summarises his view on integration analysis surveillance in haemophilia Gene Therapy. No evidence for vector related malignancy. Robust communication framework needed including how to make this data comprehensible

Some say there’s “limited interest” in #hemophilia #GeneTherapy—but the community disagrees. What PWBDs need are therapies that are effective, durable & better than what’s already available. Let’s keep pushing for real progress. Read more: bit.ly/46RoRz0