Antithrombin-lowering in hemophilia: a closer look at fitusiran

Gene therapy in haemophilia: literature review and regional perspectives for Turkey - Kaan Kavaklı, Bülent Antmen, Vahap Okan, Fahri Şahin, Selin Aytaç, Can Balkan, Ergül Berber, Zühre Kaya, Alphan Küpesiz, Bülent Zülfikar

A Molecular Revolution in the Treatment of Hemophilia: Molecular Therapy

Fitusiran, C78H139N11O30

Frontiers Hemophilia a patients with inhibitors: Mechanistic insights and novel therapeutic implications

In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

Diagram illustrating the mechanism of action of Fitusiran/ALN-AT3.

Michael Makris on X: Robert Klamroth presented the results of the phase 3 Fitusiran study. This is siRNA inhibition of antithrombin in #hemophilia with no inhibitors. 90% reduction in ABR (Primary endpoint).

Prevention and Management of Bleeding Episodes in Patients with Acquired Hemophilia A

Emicizumab, a humanized bispecific antibody to coagulation factors IXa and X with a factor VIIIa-cofactor activity

Fitusiran Reduces Bleeding in Hemophilia A, B, Regardless of Inhibitors

Fitusiran: A Game-Changing Drug for Hemophilia Patients

Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trial - The Lancet Haematology

Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial - The Lancet