An international consortium including clinicians, patient organization representatives and members of the Roche laboratory conducted a study to assess the relevance and significance of the MFM32, a functional score widely used in neuromuscular diseases and SMA in particular:
- semi-structured interviews were conducted with 40 SMA patients and 217 other patients participated in an online survey conducted in eight countries;
- the aim was to determine the significance threshold of changes as observed by the MFM32 and the patient’s own perception of a possible change;
- the MFM32 data were compared with data acquired in clinical trials;
- the study allowed the determination of thresholds of changes felt by the patient, which will have to be confirmed by complementary analyses by age and severity subgroups.
