PRO-DMD Longitudinal Natural History Study
Natural History Studies – Neuromuscular Research Center
A Prospective Natural History Study of the Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.
The purpose of this study is to look at ways of measuring how fast different muscles become weaker in children with DMD. This will provide valuable information for helping to design treatments with oligonucleotides and with other medications for DMD. There is no treatment given in this study, it is called a natural history study. The study will provide information about how to measure the progression of DMD which will, in turn, help new treatments to be developed.
There are no interventions for this study. We call these studies "Natural History Studies."
- Diagnosis of Duchenne Muscular Dystrophy resulting from a mutation in the DMD gene confirmed by a DNA diagnostics
- Age between 3 and 18 years on entry to the study
- Life expectancy of at least 3 years
- Willing and able to comply with protocol requirements
- Able to give informed consent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations).
- Current participation in a clinical study with an Investigational Medicinal Product (IMP)
- Participation within the previous 1month in a clinical study with an IMP