Hemophilia A Gene Therapy - Some Answers, More Questions

Affiliations

¹ From the Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, and the Division of Hematology and Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia - both in Philadelphia.

Editorial

Lindsey A George. N Engl J Med. 2023.

. 2023 Feb 23;388(8):761-763.

doi: 10.1056/NEJMe2212347.

¹ From the Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, and the Division of Hematology and Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia - both in Philadelphia.

No abstract available

Comment on

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
Mahlangu J, Kaczmarek R, von Drygalski A, Shapiro S, Chou SC, Ozelo MC, Kenet G, Peyvandi F, Wang M, Madan B, Key NS, Laffan M, Dunn AL, Mason J, Quon DV, Symington E, Leavitt AD, Oldenburg J, Chambost H, Reding MT, Jayaram K, Yu H, Mahajan R, Chavele KM, Reddy DB, Henshaw J, Robinson TM, Wong WY, Pipe SW; GENEr8-1 Trial Group. Mahlangu J, et al. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.