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

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.

Publication Type

Multicenter Study

Date of Publication

April 29, 2023

Journal

Lancet (London, England)

Volume/Issue

401/10386

ISSN

1474-547X

Fitusiran, a subcutaneous investigational small interfering RNA therapeutic, targets antithrombin to rebalance haemostasis in people with haemophilia A or haemophilia B, irrespective of inhibitor status. We evaluated the efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors.

Alternate Journal

Lancet

PubMed ID

37003287

Authors

Guy Young
Alok Srivastava
Kaan Kavakli
Cecil Ross
Jameela Sathar
Chur-Woo You
Huyen Tran
Jing Sun
Runhui Wu
Stacey Poloskey
Zhiying Qiu
Salim Kichou
Shauna Andersson
Baisong Mei
Savita Rangarajan

Keywords

Alanine Transaminase
Male
Humans
Adult
Hemophilia A
Young Adult
RNA, Small Interfering
Hemorrhage
Hemophilia B
Female