Duchenne is a serious condition which causes progressive muscle weakness, generally by the age of 8 boys are struggling to walk and a wheelchair is required Between 10 and 14. It is life shortening - most boys with this condition only live into their 20's and suffer from heart or respiratory failure.
Medical advancements are moving at speed (but not fast enough) and we believe that in the future the devastating effects of Duchenne will be reduced with new drugs and treatments. Several such drugs are currently on the horizon and undergoing medical trials. The current use of steroids is helping boys with Duchenne, but have serious side effects, more targeted and sophisticated treatments are being developed. Leon is young enough and has a short window of time and opportunity for these new drugs and treatments to be licensed and used to help him and other boys before they get too old and damage takes hold.