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The aim is to characterize the natural history and evaluate sensitive outcome measures for adult-onset myotonic dystrophy type 1.

August 1, 2026NeurologyOpen Access

Natural History of Adult-Onset Myotonic Dystrophy Type 1

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The aim is to characterize the natural history and evaluate sensitive outcome measures for adult-onset myotonic dystrophy type 1.
Characterized the natural history of adult-onset DM1 over 2 years.
Used quantitative MRI and clinical outcome measures including MFM32 and 6MWD.
Measured patient-reported outcomes such as DM1-ActivC and INQoL.
DM1-ActivC declined significantly at 24 months (-10.3, 95% CI -14.0 to -6.7; SRM = 0.8).
INQoL quality of life worsened at 12 months (+5.4, 95% CI 0.8-10.0) and 18 months (+6.9, 95% CI 2.0-11.8).
Distal leg PDFF correlated strongly with 6MWD (ρ = -0.81) and MFM32 D1 (ρ = -0.75).

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Randomized trial characterizes outcome measures in adult-onset myotonic dystrophy type 1, indicating sensitivity for clinical trials.

Iterbeke et al. (2026) studied this question.

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