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| Sponsor | UCL Institute of Child Health |
| Funder | AFM |
| CI | Professor Francesco Muntoni, Professor Kate Bushby |
| Sites | London GOSH, Newcastle |
| Contact details | ich.neuromusculartrials@ucl.ac.uk |
| More information |
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.
Primary objective
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
non-ambulant patients.
The tests should determine the most sensitive outcome measures for future clinical trials.