Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies

Condition(s) studied

Investigational drug(s) / intervention(s)

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.

Study 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.

Eligibility

Primary outcome measure(s)

• blood expression level of micro-RNA miR-1 — at a unique time of enrollment
  The main goal is to evaluate the interest of miR-1 as a blood biomarker for neuromuscular diseases, specifically muscular dystrophies and congenital myopathies.

Trial sites (1)

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