To access the survey please click here: https://www.research.net/s/dmd2014survey
Duchenne muscular dystrophy or DMD is the most common of the muscular dystrophies, affecting approximately 1 in every 3,500 newborn boys. It is caused by a fault in a gene called the dystrophin or DMD gene on the X chromosome. A fault in this gene stops the body making a protein called dystrophin. This protein is important in muscle fibres, and its absence results in muscle weakness that gets worse over time because muscle cells break down and are gradually lost.
While there is still no cure for DMD, it is one of the conditions where there is substantial active research and where several potential new therapies are currently being tested in clinical trials. It is also a condition for which experts have established internationally approved care guidelines that can make a big difference to the quality of life and life expectancy of a boy with the disease.
The Care-NMD survey for patients and families, will look at current treatment around Australia to identify gaps in care.
In turn, this will enable the identification of situations where best-practice recommendations are not followed, allowing for the development of future training programmes tailored to local needs. This project will also analyse the health-related quality of life (HRQL) in people with DMD and their families.