Duchenne muscular dystrophy is caused by genetic errors in the dystrophin gene which prevent the production of the essential muscle protein called dystrophin. Without this protein the muscle cells break down and die. In 1989, Professor Davies and her team discovered a protein which is very similar to dystrophin, called utrophin. In animal models, they have shown that utrophin can compensate for the absence of dystrophin. They have also set up a technique to screen for small molecules which increase the levels of utrophin. These discoveries inform this current project where Professor Davies is attempting to develop a therapy for Duchenne which compensates for the lack of dystrophin by increasing the levels of the dystrophin-related protein, utrophin. The aim of this two year project is to find small drug-like molecules which will increase the levels of utrophin in the muscle of people with Duchenne. This research is at an advanced stage and has the potential to produce a treatment for Duchenne which would halt or reduce the devastating muscle wasting associated with the condition. This would benefit the 1,800 families in the UK currently affected by Duchenne. The approach taken by the team also has wider applicability as the technique developed to screen for drugs with therapeutic benefit could also be used in other muscular dystrophies such as spinal muscular atrophy. We need £180,000 over the next three years to ensure that Professor Davies’s team can continue their search for a treatment for this devastating condition.