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In a phase 3 clinical trial, this AAV9 vector has found success in intravenously delivering the SMA1 transgeneeffectively restoring motor functions in SMA patients [157, 158]

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PSM achieved a good balance across measured covariates, and the inclusion of positive and negative control outcomes further supports internal validity

Gergs U, Boknik P, Schmitz W, Simm A, Silber RE, Neumann J (2009) A positive inotropic effect of adenosine in cardiac preparations of right atria from diseased human hearts