Trial offers hope of a treatment for spinal muscular atrophy
- 23 September 2016
Professor Matthew Wood's group has found a promising treatment for degenerative disease spinal muscular atrophy (SMA), a leading genetic cause of child death.
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 is motor neurone degeneration and increasing muscle weakness. However, people have an almost identical gene called SMN2.