Water T2 could predict functional decline in patients with dysferlinopathy

In dysferlinopathy, T2_H2O did not correlate with current functional ability. However, T2_H2O at baseline was higher in patients who worsened more rapidly on functional tests. This suggests that inter-patient differences in functional decline over time may be, in part, explained by different severities of the active muscle damage, assessed by T2_H2O measure at baseline. Significant challenges remain in standardizing T2_H2O values across sites to allow determining globally applicable thresholds. The results from the present work are encouraging and suggest that T2_H2O could be used to improve prognostication, patient selection, and disease modelling for clinical trials.