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News|Articles|September 15, 2026
Myotonic Dystrophy Awareness Day: Overviewing Emerging Therapies
Author(s)Marco Meglio
Seven experimental DM1 therapies show promise and pitfalls; see latest trial data, a major phase 3 miss, and key 2026 readouts.
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Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, caused by an expanded CTG repeat in the DMPK gene that produces a toxic RNA and disrupts normal splicing throughout the body.1 The result is a multisystem disease, marked by progressive muscle weakness and myotonia alongside cardiac, ocular, and cognitive effects, and it's more common than once thought: a 2021 population-based analysis put its prevalence at roughly 1 in every 2,100 people.2 There is still no FDA-approved disease-modifying therapy.
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