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Myotonic Dystrophies: Targeting Therapies for Multisystem Disease
Myotonic Dystrophy (DM) is an inherited, multisystemic disorder that affects muscle and various other organs. There are two types: DM1 and DM2. DM1, caused by CTG repeat expansion in the "DMPK" gene, is more common and severe, involving muscle weakness, myotonia, and systemic complications such as cardiac, respiratory, and gastrointestinal issues. DM2, caused by CCTG repeat expansion in the "CNBP" gene, presents with less severe systemic symptoms but is still …
