inStem (Institute for Stem Cell Science and Regenerative Medicine)

Department of Biotechnology
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

Journal Article

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