SMA is a genetic neuromuscular disease, which means it is inherited and affects nerves responsible for muscle function. Although classified as rare, SMA is the leading genetic cause of death of infants and toddlers, with approximately 95% of the most severely diagnosed cases resulting in death by the age of 18 months. Children with a less severe form of SMA face the prospect of progressive muscle wasting, loss of mobility and motor function.

1 in 40 to 60 people is an SMA carrier, i.e. approximately 1.5 million people in the UK

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