Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A - Mahlangu, Johnny; Kaczmarek, Radoslaw; von Drygalski, Annette; Shapiro, Susan; Chou, Sheng-Chieh; Ozelo, Margareth C.; Kenet, Gili; Peyvandi, Flora; Wang, Michael; Madan, Bella; Key, Nigel S.; Laffan, Michael; et al. (The New England journal of medicine, 2023)
Keywords: Bleeding; Body weight; Coagulation; Coagulation factors; Confidence intervals; Disease prevention; Epidemiology; Factor VIII - therapeutic use; Factor VIII deficiency; Gene transfer; Gene Transfer Techniques; Genetics; Genetics General; Genomes; Half-Life; Hematology; Hemophilia; Hemophilia A - complications; Hemophilia A - drug therapy; Hemorrhage - etiology; Hemorrhage - prevention & control; HIV; Human immunodeficiency virus; Humans; Hypotheses; Life Sciences; Male; Oncology; Pharmacokinetics; Prophylaxis; Recombinant Fusion Proteins - therapeutic use; Safety; Statistical analysis