In the last three decades, gene therapy has demonstrated significant progress. Over 700 active investigational new drug (IND) applications have been reported. Research on in utero gene therapy has advanced, but ethical and safety concerns persist.
View Article and Find Full Text PDFObjective: Disease severity scores, or endpoints, are routinely measured during Randomized Controlled Trials (RCTs) to closely monitor the effect of treatment. In real-world clinical practice, although a larger set of patients is observed, the specific RCT endpoints are often not captured, which makes it hard to utilize real-world data (RWD) to evaluate drug efficacy in larger populations.
Methods: To overcome this challenge, we developed an ensemble technique which learns proxy models of disease endpoints in RWD.
Women with primary mitral insufficiency have a smaller regurgitant volume at the same regurgitant fraction than men. We hypothesized that normalizing regurgitant volume with left ventricular end-diastolic volume or allometric scaling would eliminate the difference in regurgitant volume between women and men. The study cohort consisted of 101 patients with mitral valve prolapse undergoing cardiac MRI.
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