SMA is caused by a faulty gene that affects nerve cells needed to control the muscles we use for moving, swallowing and breathing. 1 in 40 of us is a carrier and in the most severe cases (50% of those affected), children rarely live beyond 2 years old – making SMA the leading genetic cause of death in babies and toddlers.
The main aims of the consortium are to further develop existing drug targets and identify new neuroprotective therapies to maintain nerve function throughout the lives of people living with SMA, and to identify improved ways of delivering treatments in order to maximise benefit throughout the body.