Because gene therapies are used to treat diseases affecting small numbers of patients, “they are approved by the [European Medicines Agency (EMA)] and FDA with
Two years after gene transfer using valoctocogene roxaparvovec, recipients had sufficient factor VIII activity and bleeding reduction, a study shows. “Models of the risk of
A clinical trial of etranacogene dezaparvovec gene therapy demonstrates its superiority over prophylactic factor IX with respect to annualized bleeding rate, researchers report, with a
Artemis-deficient severe combined immunodeficiency (ART-SCID) in newly diagnosed infants responded to an infusion of lentiviral gene-corrected autologous CD34+ cells, a study shows. Artemis, a DNA-repair
Clinical studies reported in the current issue of the New England Journal of Medicine include a phase 3 trial of beremagne geperpavec (B-VEC) in patients
FDA yesterday approved etranacogene dezaparvovec (Hemgenix, CSL Behring), an adeno-associated virus vector-based gene therapy for the treatment of adults with hemophilia B (congenital factor IX
FDA is alerting medical device users about a cybersecurity risk for the Medtronic MiniMed 600 Series Insulin Pump System, including the MiniMed 630G and MiniMed