Neuromuscular diseases (NMDs) are in many cases genetic and rare disorders affecting patients from their young age and progressing over life. The most severe NMDs are associated with reduced life expectancy and poor quality of life. As NMDs often lead to forms of disability, the burden to families, carers and society is usually very high. At forethought access we assess and explore potential for new therapies to improve patient outcomes while offsetting healthcare and societal costs due to NMDs, therefore creating added value for healthcare organizations.