One low dose, lasting effect: a new RNA approach shows promise for Duchenne in the lab

In laboratory models of Duchenne muscular dystrophy — a condition that weakens muscles from childhood — a single low dose of Utrecht-based Sapreme's experimental treatment restored the missing muscle protein dystrophin to levels researchers consider meaningful, with effects that lasted. The early, preclinical results were presented at a scientific meeting in Denver; human testing is still ahead, but the approach aims for stronger effects with less frequent dosing than today's options.