Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: Final phase 3 GENEr8-1 trial results

Authors

• Andrew D Leavitt, University of California, San Francisco
• Elaine Majerus, Washington University School of Medicine in St. Louis
• et al.

Journal

Research and Practice in Thrombosis and Haemostasis

Publication Date

3-1-2026

Volume

10

Issue

3

First Page

103416

Document Type

Open Access Publication

DOI

10.1016/j.rpth.2026.103416

Rights and Permissions

Leavitt AD, Mahlangu J, Raheja P, Symington E, Quon DV, Giermasz A, Kenet G, Lowe G, Key NS, Millar CM, Pipe SW, Chou SC, Klamroth R, Mason J, Chambost H, Peyvandi F, Majerus E, Pepperell D, Tan CW, Yu H, Chavele KM, Ozelo MC. Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results. Res Pract Thromb Haemost. 2026 Mar 27;10(3):103416. doi: 10.1016/j.rpth.2026.103416. © 2026 The Author(s). Published by Elsevier Inc. on behalf of International Society on Thrombosis and Haemostasis. This is an open access article under the CC BYNC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

Recommended Citation

Leavitt, Andrew D; Majerus, Elaine; and et al., "Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: Final phase 3 GENEr8-1 trial results." Research and Practice in Thrombosis and Haemostasis. 10, 3. 103416 (2026).
https://digitalcommons.wustl.edu/oa_4/7264

Department

ICTS (Institute of Clinical and Translational Sciences)

Additional Links

Supplemental material is available for this article at publisher site.