Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials

Schreiber A, Brochard S, Rippert P, Fontaine-Carbonnel S, Payan C, Poirot I, Hamroun D, Vuillerot C 
Dev Med Child Neurol. 2018. 60(2):185-191.
The aim of the study was to monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients.
Significant differences were found between mean MFM D1, D2 and D3 scores in corticosteroids-treated and untreated groups bewteen 0 and 6 months and between 0 and 24 month. For MFM D1 specifically, there was a significant increase in the corticosteroids-treated group (mean slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (mean slope of change=-17.8±17.7%/y) between 0 months and 6 months.

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Keywords: Duchenne muscular dystrophy, corticosteroids, Motor Function Measure

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