Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy

Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy

There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration. Although the relevance of that rule to exercises in neuromuscular patients and for SMA in particular is not definitely proven, clinical observations seem to support this assumption. Also there are several experimental studies which provide additional support for utility of exercise for SMA.

However, making regular exercises may be very challenging with SMA not only due to physical limitations, but due to psychological either.

While being considered as safe and well tolerated intervention, TMS is able to mimic effects of real physical exercises, at least at the level of low motoneuron, it also provides several advantages. For example, possibility to exercise non-collaborative infants, minimization of psychological motivation impact in adults and/or ability to involve very weak muscle groups.

Inclusion Criteria:

• Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by DNA test.
• Informed consent.
• A minimum score of 1 for Entry Item "A" of the Revised Upper Limb Module (RULM) scale for SMA: "Can use hands to hold pencil or pick up a coin/token or drive a powered chair, use phone key pad"

Exclusion Criteria:

• Subject has severe joint contractures that would affect ability to perform study measures, determined by the study physician.
• Subject has a deconditioned respiratory system, per the discretion of the physician investigator.
• Subject has behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator.