Cytokinetics/ Astellas CK-2127107

Lower motor neurons of the spinal cord are the main cell type affected by SMA. When the lower motor neurons deteriorate, they no longer efficiently contact their target muscles, ultimately leading to loss of voluntary muscle contraction, which causes muscle wasting, or atrophy, due to inactivity.

The goal of muscle protection is to protect the muscles from atrophy, increase muscle mass, and perhaps even restore some muscle function. Similar to neuroprotection, this strategy does not address the underlying genetics of SMA, rather, the aim is to slow or stop the progression of the disease. It could be used in combination with approaches designed to increase SMN protein levels.

It is thought that these approaches may be more appropriate for milder types of SMA. They may include:

Ongoing muscle protective trials for SMA

Tirasemtiv is being studied in clinical trials for people with MotorNeurone Disease (MND) by Cytokinetiks. The effects of Tirasemtiv on leg and respiratory muscle function are being examined and whether it may reduce fatigue and improve muscle strength during exercise. As motor neurons are affected in both MND and SMA, the possibilities are also being investigated for SMA.