Although ocular signs in LEMS are typically mild and appear late, recognizing them alongside proximal weakness and autonomic symptoms can speed diagnosis and earlier detection of an associated lung cancer.
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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