In this cross-sectional study, approved label populations were broader overall compared to trial populations for new drugs in ...

In this prospective cohort study, a single intravenous dose of scAAV2/8-LP1-hFIXco gene therapy in men with severe hemophilia B led to sustained factor IX expression and significantly reduced bleeding ...

In this retrospective cohort study, the presence of tumor-infiltrating clonal hematopoiesis (TI-CH) was associated with an ...

Type 2 diabetes and abnormal amyloid-β concentration (low Aβ42:Aβ40 ratio) in the cerebrospinal fluid were associated with accelerated brain atrophy and earlier progression from normal cognition to ...