The variations of the MFM32 seen by the patient with SMA

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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.

 

A Patient-Centered Evaluation of Meaningful Change on the 32-Item Motor Function Measure in Spinal Muscular Atrophy Using Qualitative and Quantitative Data. Duong T, Staunton H, Braid J, et al. Front Neurol. 2022 Jan 17;12:770423.

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