Ionis Molecule Shows Early Promise for Myotonic Dystrophy Type 1

Ionis Molecule Shows Early Promise for Myotonic Dystrophy Type 1

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Ionis Molecule Shows Early Promise for Myotonic Dystrophy Type 1

High levels of the toxic RNA molecule associated with myotonic dystrophy type 1 (DM1) lead to defects in muscle regeneration and in the number of muscle stem cells — or satellite cells — and promotes muscle fibrosis (scarring) upon injury, according to a study in a mouse model of DM1. IONIS 877864, Ionis Pharmaceuticals’ investigational therapy targeting this toxic RNA molecule, was found to effectively correct these defects and prevent further muscle damage and fibrosis. These…

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