Department of Biotechnology
inStem (Institute for Stem Cell Science and Regenerative Medicine)

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

Publication Type

Journal Article

Date of Publication

May 1, 2023

Journal

The Lancet. Haematology

Volume/Issue

10/5

ISSN

2352-3026

Fitusiran, a subcutaneous investigational siRNA therapeutic, targets antithrombin with the goal of rebalancing haemostasis in people with haemophilia A or haemophilia B, regardless of inhibitor status. We aimed to evaluate the efficacy and safety of fitusiran prophylaxis in people with severe haemophilia without inhibitors.

Alternate Journal

Lancet Haematol

PubMed ID

37003278

Authors

Alok Srivastava
Savita Rangarajan
Kaan Kavakli
Robert Klamroth
Gili Kenet
Liane Khoo
Chur-Woo You
Weiqun Xu
Niel Malan
Laurent Frenzel
Catherine N Bagot
Oleksandra Stasyshyn
Chia-Yau Chang
Stacey Poloskey
Zhiying Qiu
Shauna Andersson
Baisong Mei
Steven W Pipe

Keywords

Hemophilia B
Male
Humans
Hemophilia A
RNA, Small Interfering
Hemorrhage
Blood Coagulation Factors