Prepare the Way for Hemophilia A Gene Therapy

Affiliations

¹ From the Hemophilia and Thrombosis Treatment Center, Rady Children's Hospital San Diego, San Diego, and the Department of Pediatrics, University of California San Diego Health Sciences, La Jolla.

Editorial

Courtney D Thornburg. N Engl J Med. 2022.

. 2022 Mar 17;386(11):1081-1082.

doi: 10.1056/NEJMe2200878.

¹ From the Hemophilia and Thrombosis Treatment Center, Rady Children's Hospital San Diego, San Diego, and the Department of Pediatrics, University of California San Diego Health Sciences, La Jolla.

No abstract available

Comment on

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
Ozelo MC, Mahlangu J, Pasi KJ, Giermasz A, Leavitt AD, Laffan M, Symington E, Quon DV, Wang JD, Peerlinck K, Pipe SW, Madan B, Key NS, Pierce GF, O'Mahony B, Kaczmarek R, Henshaw J, Lawal A, Jayaram K, Huang M, Yang X, Wong WY, Kim B; GENEr8-1 Trial Group. Ozelo MC, et al. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.