What type of study is this?

This is a Human Clinical Trial study.

What question did this study set out to answer?

To establish reliable biomarkers and clinical endpoints for myotonic dystrophy type 1 by studying disease progression and phenotypic heterogeneity.

December 11, 2025PLoS ONEOpen Access

Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study

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To establish reliable biomarkers and clinical endpoints for myotonic dystrophy type 1 by studying disease progression and phenotypic heterogeneity.
Prospective, multi-site observational study
Involves approximately 700 adult DM1 patients
Includes strength testing, myotonia assessment, spirometry, and cognitive tests
Collects blood and urine samples for biomarker studies
Follows up over 24 months with visits at baseline, 12, and 24 months
Aims to characterize phenotypic heterogeneity and disease progression of DM1
Seeks to identify baseline characteristics predicting progression
Focuses on validating RNA biomarkers related to disease severity

Discussion

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Implication

International study reveals baseline characteristics predicting disease progression in myotonic dystrophy type 1, highlighting the importance of reliable biomarkers.

Cite This Study

Mul et al. (2025) studied this question.

synapsesocial.com/papers/69401b1e2d562116f28f75ce https://doi.org/10.1371/journal.pone.0331163

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Implication

International study reveals baseline characteristics predicting disease progression in myotonic dystrophy type 1, highlighting the importance of reliable biomarkers.

Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study

Key Points

Discussion

Implication

Cite This Study

Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study

Key Points

Discussion

Implication

Cite This Study

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