French clinicians report the development and results of a follow-up protocol for infants with SMA treated with gene therapy (onasemnogene abeparvovec or Zolgensma®):
- 23 children with SMA, including 19 with type I, 3 with type II and one presymptomatic, were included in this observational study,
- a device was used to calculate the inertial measurement units (IMU) of several target joints (wrists, elbows and ankles) as a function of the child’s supine movements, first spontaneously and then in the presence of a toy,
- the evaluation was carried out at regular intervals (M0, M1, M3, M6, M12, M18 and M24) and showed improvements in motor skills, particularly in the elbow sensor, demonstrating the effectiveness of Zolgensma®.
Such an approach could be incorporated into the therapeutic trials currently being developed for SMA.
