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Häm ist ein Cofaktor mit unzähligen Funktionen, der für fast alle lebenden Organismen essentiell ist. Neben dem klassischen Transport von Gasen und katalytischen Funktionen wird Häm zunehmend als streng kontrolliertes Signalmolekül wahrgenommen, das die Proteinexpression reguliert. Häm‐Akquisition, ‐Biosynthese und ‐Regulation sind jedoch nur in wenigen Modellorganismen gut erforscht und darüber hinaus...
Duchenne muscular dystrophy (DMD) is a fatal muscle‐wasting disease arising from mutations in the dystrophin gene. Upregulation of utrophin to compensate for the missing dystrophin offers a potential therapy independent of patient genotype. The first‐in‐class utrophin modulator ezutromid/SMT C1100 was developed from a phenotypic screen through to a Phase 2 clinical trial. Promising efficacy and evidence...
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