55
Participants
Start Date
June 24, 2025
Primary Completion Date
June 24, 2027
Study Completion Date
June 24, 2028
Quantitative neuromuscular MRI
Quantification of biomarkers as fat fraction, magnetization Transfer Ratio, muscular volume, relaxation time T2
Skin biopsy
Performed on the arm or index finger, depending on patient age
Clinical scores
ONLS, CMTES-R, CMT-Peds, CMT-FOM
Blood test
10 ml sample
Patient Report Outcomes Measures
pCMT-QoL, EVA, WALK-12, PGI-c, SF-12
RECRUITING
Assistance Publique - Hôpitaux de Marseille, Marseille
Association Française contre les Myopathies (AFM), Paris
OTHER
University Medical Center Göttingen
UNKNOWN
Assistance Publique Hopitaux De Marseille
OTHER