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Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
Sponsor: University Hospital, Clermont-Ferrand
Summary
The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases. MicroRNAs (miRs) are small messengers that help control how cells grow and stay healthy. Some of these, like miR-1, are specifically found in muscles and the heart. Research shows that levels of miR-1 are often abnormal in people with muscle-wasting conditions, but more information are needed to understand how this relates to the severity of the disease. The main goal is to compare the blood levels of miR-1 between four different groups at different ages and severities: 1. Patients with Duchenne or Becker muscular dystrophy (DMD/DMB). 2. Patients with Myotonic Dystrophy Type 1 (Steinert's disease). 3. Patients with congenital myopathies. 4. Healthy volunteers (control group). The main objective is to assess if miR-1 levels can accurately show how a muscular disease is progressing.
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
INTERVENTIONAL
Enrollment
104
Start Date
2026-02-11
Completion Date
2029-03
Last Updated
2026-02-25
Healthy Volunteers
Yes
Conditions
Interventions
dosage of blood biomarker miR1
Patients and controls will be asked to provide blood samples to evaluate their blood level of miR1 biomarker on a unique time participation.
Locations (1)
CHU de Clermont-Ferrand
Clermont-Ferrand, France