Duchenne Muscular Dystrophy Clinical Trial
Official title:
Quantification of Muscle Specific microRNAs in the Serum of Patients With Duchenne Muscular Dystrophy (DMD) and Becker (BMD) : Evaluation of the Inters-est of These Biomarkers in Patients Care
Duchenne muscular dystrophy (DMD) , caused by mutations in the DMD gene, is the most common
and most severe progressive dystrophy of the child. Although the development is rapidly
progressive , there is variability in the severity of the disease between DMD patients that
do not correlate with the type of mutations in the DMD gene. There are no easily measurable
biomarkers for monitoring the DMD or moderate form of the disease, Becker muscular dystrophy
(BMD ) . MicroRNAs (miRNAs) are involved in most cellular processes , and their expression
pattern is a signature of the state of a cell . They represent a potential class of
diagnostic and prognostic biomarkers. Some are specific for the skeletal myogenesis , and
changes in their pattern of expression are associated with muscle diseases including muscular
dystrophy. The levels of muscle- specific miRNAs are indeed greatly increased in the serum of
DMD and BMD compared to control patients .
The main objective of this is to validate the use of serum muscle-derived microRNAs as
biomarkers of DMD patients (compared with healthy subjects). Secondary objectives are i) to
investigate the relationship between circulating levels of these miRNAs and the severity of
the dystrophinopathy (DMD vs BMD) and also the progression of the disease (longitudinal
study), ii) to assess the specificity of these markers for dystrophinopathy (comparison with
other patients with muscular dystrophy), iii) to test candidate miRNAs recently identified
but not yet analyzed in the serum of patients.
Clinical data and samples will be recorded at each regular consultation. miRNA levels will be
quantified using Real Time Quantitative RT-PCR.
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