Last day starts with an
Inspiring presentation @rkaczmarekdr.bsky.social #ISTH2025 #hemophilia #genetherapy #Toleranceinduction
Will gene therapy be used for tolerance induction in future?

What percentage of hemophilia patients develop inhibitors?

New data from the European Haemophilia Safety Surveillance Inhibitor reveals inhibitor rates of
Hemophilia A: 4.2 per 1000 treatment years
Hemophilia B: 0.1 per 1000 treatment years

Read more here: www.rpthjournal.org/article/S247...

Nice #glycotime review that pulls together the emerging story of glycosyltransferase specificity for cellular substrates

chemistry-europe.onlinelibrary.wiley.com/doi/10.1002/...

Specificity in glycosylation! Have you ever wondered why some glycosyltransferases recognize glycoproteins, some glycolipids, and some both? Together with @kmikolajczyk.bsky.social and @rkaczmarekdr.bsky.social, we try to answer that complicated question.

#glycotime
tinyurl.com/5b6kxhp3

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

Comprehensive review of gene therapy in #hemophilia accepted for publication by Molecular Therapy. The pre-proof is on line at tinyurl.com/3sb4nuex

Our paper on inhibitor trends is now published in the journal Haemophilia. The number of inhibitors in previously untreated patients has markedly reduced, but it is possible that this is due to delayed exposure to FVIII. tinyurl.com/2mr3t5e3

I was pleased to moderate the session on basic and translational research in coagulation and fibrinolysis with Dr. Carlos Bravo-Perez at #ASH2024 earlier today.

Six excellent presentations are now available on the Virtual Platform.