Queen Square Centre for Neuromuscular Diseases


Outcome measures in Duchenne Muscular Dystrophy (DMD): A Natural History Study

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.