Spinal Muscular Atrophy – final stages of development of a treatment

In the Sheffield Institute for Translational Neuroscience (SITraN), the gene therapy group led by Prof Mimoun Azzouz is approaching the final stages of preclinical development of a treatment for Spinal Muscular Atrophy (a childhood disease of the motor neurones). Here Chris Binny explains more about the group’s work.

About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an inherited disease affecting between 1/6,000 and 1/10,000 newborns. These patients have inherited two faulty copies of the SMN1 gene and, as a result, can’t make enough of the protein “survival of motor neuron” or SMN. This causes the gradual loss of motor neurons, causing progressive muscle weakness and paralysis beginning in the lower legs and spreading upwards over a period of months or years. Read More »