Animal models of spinal muscular atrophies: Towards a better understanding of pathophysiology and development of therapeutics

[Autom. eng. transl.] Spinal amyotrophies are neuromuscular diseases characterized by motor neuron degeneration associated with muscle paralysis and atrophy. The identification of the responsible gene (SMN), the knowledge of the genetic basis of a severe or moderate form of the disease, a better understanding of the function of SMN and the production of animal models have allowed to identify several strategies of therapeutic research. These can be undertaken on the developed models.