Exon 45 Skipping Therapy for DMD Shows Safety in Small Trial

Exon 45 Skipping Therapy for DMD Shows Safety in Small Trial

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Exon 45 Skipping Therapy for DMD Shows Safety in Small Trial

A potential exon-skipping therapy for Duchenne muscular dystrophy (DMD) called DS-5141 showed a good safety profile with repeat dosing in a small clinical trial, according to a press release. The Phase 1/2 clinical trial (NCT02667483) tested DS-5141 for a first time in Duchenne patients amendable to exon 45 skipping. No safety concerns, including clinically significant adverse events, were reported, and no one stopped treatment early. DMD is caused by mutations in the DMD gene, which encodes the instructions necessary to produce…

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