The New England journal of medicine
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Multicenter Study
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.
Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX-Padua), was associated with sustained factor IX activity in a phase 1-2a study. ⋯ Fidanacogene elaparvovec was superior to prophylaxis for the treatment of participants with hemophilia B, leading to reduced bleeding and stable factor IX expression. (Funded by Pfizer; BENEGENE-2 ClinicalTrials.gov number, NCT03861273.).
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Randomized Controlled Trial
Results after Four Years of Screening for Prostate Cancer with PSA and MRI.
Data on the efficacy and safety of screening for prostate cancer with magnetic resonance imaging (MRI) are needed from studies of follow-up screening. ⋯ In this trial, omitting biopsy in patients with negative MRI results eliminated more than half of diagnoses of clinically insignificant prostate cancer, and the associated risk of having incurable cancer diagnosed at screening or as interval cancer was very low. (Funded by Karin and Christer Johansson's Foundation and others; GÖTEBORG-2 ISRCTN registry number, ISRCTN94604465.).