Perspectives on models of spinal muscular atrophy for drug discovery

Spinal muscular atrophy (SMA) is a progressive motor neuron disease, which is one of the most common forms of inheritable infant death in the world. SMA results from reduced levels of SMN protein. Various models systems used in SMA research are available and it is possible to assess their strengths and weaknesses. Although each of these models has its limitations, they must be used together so as to effectively and efficiently identify potential SMA therapeutics.