Active clinical trials for "Muscular Dystrophies"

Facioscapulohumeral muscular dystrophy (FSHD) is the second most common muscular dystrophy with approximately 500 patients in Denmark. It is characterized by weakness and wasting of the facial muscles, the muscles in the shoulder region and of the legs. The primary aim of this study is to investigate possible links and order between inflammation and fat infiltration in the muscles in patients with FSHD. Approximately 15 patients with FSHD will be recruited for repeated MRI-scans during a year where the inflammation and fat infiltration in the muscles can be quantified.

Family-centred care (FCS) is considered the best practice in providing rehabilitation to children with disabilities and special needs. Family-centred care has been described as a partnership approach to healthcare decision making. As a philosophy of healthcare, today many multidisciplinary healthcare facilities have organized their services according to a family-centred approach. TheMeasure of Processes of Care (MPOC) is the most widely used instrument to assess parents' self-reported experiences of family-centred behaviours of rehabilitation services providers. The aim of this study is to translate the scale to Turkish and to determine validity and reliability of The Measure of Processes of Care (MPOC 56- 20- SP)

There isn't specific Health related quality of life measure for children with DMD in French. The aim of this study is to validate the French version of the Pediatric Quality of Life Inventory 3.0 Duchenne Muscular Dystrophy module with a multicentric study. The investigators will evaluate the following psychometric properties : convergent validity, internal validity, inter-rater reliability. The investigators would like to be able to use this scientific tool in future clinical trials.

This study will have significant impact on muscular dystrophy patients as it promotes early screening for heart disease. With early identification, beneficial medical therapy can be started sooner, resulting in restoring and maintaining normal heart function. This is critical to the survival of these patients. We have reported previously that heart failure in all patients may have common mechanisms, the "final common pathway". Heart failure is a significant health problem with 5 million people in the US carrying the diagnosis and accounting for 12-15 million office visits and 6.5 million hospital days per year. The number of deaths from heart failure continues to increase. The data from this study could impact patients worldwide with heart failure by offering new insight into an ever-growing disease population and lead to significant changes in how they are currently treated.

This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic resonance imaging.