DMD/BMD – OUTCOME MEASURES

Duchenne muscular dystrophy (DMD) is a rapidly progressive X-linked disorder characterized by muscle weakening responsible for the loss of ambulation around age 12. One if limiting factor clinical development sensitivity and reliability current outcomes. Recently, 95th centile stride velocity (SV95C) was qualified European medicines agency as valid secondary outcome trials in subjects with DMD. Gathering normative data crucial to pursue validation process. For this purpose, 91 healthy volunteers aged from 6 85 years were enrolled ActiLiège study. Eighty-four assessed at baseline 12 months later. They performed 6-minute walk, 4-stair climb, rise floor, 10-metre walk tests. one month 1-year, participants also asked wear ActiMyo®, wearable device designed accurately passively capture limb movements daily living. Afterwards, length, distance walked per hour calculated over each recording period. At baseline, SV95C 2.6 m/s ± 0.4 children 1.6 0.3 adults. It not correlated height or children, did significantly change 1-year 6MWT, 4SC 10MWT it In we observed significant correlations length (50th centile) (ρ=0.869, ρ=0.312) (ρ=0.876, ρ=0.274). These measures increase timed tests (6-minute tests). Those permit identify variables important confounding factors help interpret longitudinal evolution time patient populations.