SMA occurs when people lack a gene called survival motor neuron 1 (SMN1). It can affect children in the womb or adults. This makes them unable to produce enough SMN protein, resulting in motor neurone degeneration and increasing muscle weakness. However, people have an almost identical gene called SMN2.
Trial offers hope of a treatment for spinal muscular atrophy
20 September 2016
A research team led by the University of Oxford has found a promising treatment for degenerative disease spinal muscular atrophy (SMA), a leading genetic cause of child death.