We are partnering with people living with spinal muscular atrophy (SMA) and patient organisations to explore multiple approaches to treatment that help improve lives.
SMA is an autosomal recessive genetic disorder caused by reduced levels of SMN protein throughout the body, resulting from mutations in the survival motor neuron-1 (SMN1) gene. It affects approximately 1 in 10,000 babies born worldwide each year and is the most common genetic cause of death in infants.
It is a disease that robs people of their physical strength by affecting motor nerve cells, taking away the ability to walk, eat and even breathe.
We have been inspired by the SMA community throughout our ten-year history of research and investigation into the SMA. Working together, we continue to strive for further understanding and progression of the science behind SMA.