Seeking boys aged 5-9 years of age who have been diagnosed with Duchenne Muscular Dystrophy (DMD) to learn more about the natural course of the disease.
The aim of this study is to better understand Duchenne Muscular Dystrophy (DMD) in young boys by recording very accurately specific data related to Duchenne disease and, therefore, to better understand and anticipate the disease course evolution. Participating children will be followed regularly to assess in particular his muscular, cardiac and respiratory symptoms, but also their quality of life, with dedicated tests and scales.
What we're hoping for
To learn more about the course children's Duchenne Muscular Dystrophy (DMD). Such a study is particularly important in the perspective of future therapeutic clinical studies that might be proposed to people presenting Duchenne disease.
ClinicalTrials.gov Identifier: NCT03882827