Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.
Cuker A, Kavakli K, Frenzel L, Wang JD, Astermark J, Cerqueira MH, Iorio A, Katsarou-Fasouli O, Klamroth R, Shapiro AD, Hermans C, Ishiguro A, Leavitt AD, Oldenburg JB, Ozelo MC, Teitel J, Biondo F, Fang A, Fuiman J, McKay J, Sun P, Rasko JEJ, Rupon J; BENEGENE-2 Trial Investigators.
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N Engl J Med. 2024 Sep 26;391(12):1108-1118. doi: 10.1056/NEJMoa2302982.
N Engl J Med. 2024.
PMID: 39321362
Clinical Trial.