In order to define parameters for monitoring and evaluating Becker muscular dystrophy (BMD) that could be used in clinical trials, a Belgian team conducted a study in 21 BMD patients over 18 months.
- Changes in muscle magnetic resonance imaging (MRI), patient-reported criteria (ActivLim, SF36), numerous clinical evaluation criteria, including motor function measurement (MFM), muscle strength and timed tests were assessed at inclusion, 9 and 18 months.
- MRI was sensitive enough to detect a significant increase in thigh muscle fat fraction as early as nine months.
- MFM-32, North Star Ambulatory Assessment (NSAA) and ActivLim scores had all deteriorated significantly by nine months.
- MFM domain 1 showed the greatest sensitivity in detecting motor changes.
