The objective of the study is to identify the best clinical and biological outcome measures for further therapeutics approaches.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
NONE
Enrollment
20
Institute of Myology
Paris, France
Measure of changes in motor function and muscle strength assessment between 2 time points
Time frame: baseline, 1 year, 2 years, 3 years
Measure of changes in NMR upper and lower limbs assessment between 2 timepoints(not for controls)
Time frame: baseline, 1 year, 2 years, 3 years
24h urine and serum collection, measures at different time points
Time frame: baseline, 1 year, 2 years, 3 years
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