The Fund has three clear goals:
We believe that investigating a variety of therapeutic approaches will be necessary in order to develop effective treatments for people with Duchenne muscular dystrophy. Professor Dame Kay Davies says:
There are so many exciting approaches to therapy at the moment. Combination therapy might be additive because each treatment targets different pathways. One drug reducing the inflammatory response for example may enable other therapies such as exon skipping and utrophin upregulation to work more efficiently.
The North Star natural history project, patient registries and clinical trials co-ordinators all need to be in place in advance, co-ordinated and well managed, to ensure clinical trials can take place in the UK.
New treatments may carry a relatively high cost for health commissioners as these are classed as ‘rare conditions’ and Muscular Dystrophy UK has the influencing skills and ability to ensure that patients have early access to any emerging treatments.
Following our high profile campaign for access to Translarna – the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy – the National Institute for Health and Care Excellence (NICE) has published guidance recommending funding on the NHS in England for Translarna. Translarna has also been approved in Northern Ireland, and children in Scotland can expect to receive the drug on an individual funding basis. The Welsh Government is yet to make an announcement, but it is hoped that they will approve the drug as it has been approved by NICE. The Isle of Man Government has rejected funding for the drug, and Muscular Dystrophy UK is working to help get the decision reversed.
Support the Duchenne Research Breakthrough Fund now.