Therapeutic developments for Duchenne muscular dystrophy

Nature Reviews Neurology, Published online: 30 May 2019; doi:10.1038/s41582-019-0203-3Verhaart and Aartsma-Rus discuss important advances in the treatment of Duchenne muscular dystrophy. Gene-addition, exon-skipping, stop codon readthrough and genome-editing approaches aim to restore expression of partially functional dystrophin, whereas treatments that target disrupted pathophysiological pathways can improve muscle function.
Source: Nature Reviews Neurology - Category: Neurology Authors: Source Type: research