G.P.167: Clinical trial readiness for non-ambulatory boys and men with Duchenne muscular dystrophy: 12 and 24 month follow-up from the MDA–DMD Network

Abstract

Few clinical trials involve non-ambulatory boys and men with Duchenne muscular dystrophy. We previously tested clinical outcomes in 91 subjects and found Forced Vital Capacity (FVC), Brooke and Egen Klassifikation (EK) Scales, grip, key and pinch strength showed excellent intra-rater reliability and feasibility. We then followed subjects every six months. Over the two-year study, eight died (mean 18.7; range 33–12 years) and 33 missed at least one visit or dropped out. Four of the eight who died were taking corticosteroids and four were not. Sixty-six completed baseline and 12-month follow-up; 51 completed baseline and 24-month follow-up; 39 completed all 5 visits. FVC and and grip strength both declined significantly over one and two years (two tailed p for all