|Sponsor||UCL Institute of Child Health|
|CI||Professor Francesco Muntoni, Professor Kate Bushby|
|Sites||London GOSH, Newcastle|
This is a 4 years follow-up study in ambulant and
non-ambulant patients. This prospective longitudinal natural history study will
be performed in two groups of DMD patients.
One group is ambulant patients and the second non-ambulant. Inclusion criteria and methods will be different in the two groups.
To document with quantified measurements the natural history of DMD.
Several validated tools will be used to describe:
- motor, orthopaedic and respiratory functions
- quality of life
- and blood parameters
Secondary objectives are specific tests to ambulant and
The tests should determine the most sensitive outcome measures for future clinical trials.