What type of study is this?

This is a Observational study (also classified as: Human Clinical Trial, Pre-Registered Trial).

August 19, 2025Open Access

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1): protocol of an international natural history study

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Identifying clinical endpoints may enhance the design of clinical trials for myotonic dystrophy type 1.
In this study, approximately 700 adult DM1 patients will be observed over a 24-month follow-up period.
A multi-site observational study will assess the phenotypic heterogeneity and the role of RNA biomarkers.
Results will provide vital data to support future research efforts in myotonic dystrophy type 1.

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Implication

Observational analysis examines disease progression and biomarkers in 700 adults with myotonic dystrophy type 1, highlighting critical insights for clinical trials.

Cite This Study

Mul et al. (2025) studied this question.

synapsesocial.com/papers/68af4eaead7bf08b1ead7353 https://doi.org/10.1101/2025.08.13.25333647

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Implication

Observational analysis examines disease progression and biomarkers in 700 adults with myotonic dystrophy type 1, highlighting critical insights for clinical trials.

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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