Kate Bushby, a leading professor of neuromuscular genetics, has been leading a clinical trial of Translarna at The John Walton Muscular Dystrophy
Research Centre, which is part of the Institute of Genetic Medicine at Newcastle University.
is a condition where the muscles gradually weaken.
Since no new drug development would be needed, repurposing it for muscular dystrophy
could quickly transform clinical practice.
A review of the Muscular Dystrophy
products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
It is a slowly progressive dystrophy
which appears as multiple, tiny epithelial vesicles which extend to the limbus and are most numerous in the interpalpebral area, with clear surrounding epithelium.
However, until now, it was not clear whether molecular abnormalities similar to those in muscle tissue of individuals with mytonic dystrophy
also occur in the brain, resulting in the cognitive neurological problems.
Three to four boys are diagnosed with Duchenne muscular dystrophy
as a result of the screening programme every year in Wales, although the same number of families also receive false positive results.
For more details about the Big Fun Run please email the Muscular Dystrophy
Campaign Regional Office at northwest@muscular-dystrophy
After seeing the Muscular Dystrophy
Campaign's advertisement, it seemed like a great opportunity to fulfil a lifelong dream - and for such a good cause.
The six-year-old from Marston Green, who has Duchenne muscular dystrophy
, will be seen on more than 1,000 sites across the country.
As Tesco Charity of the Year 2009, the Muscular Dystrophy
Campaign is aiming to raise pounds 3m to provide vital, specialist equipment such as powered wheelchairs and electric beds, for the 8,000 children in the UK who have a muscle-wasting disease.
If you think you can spare the time, please contact Laura White at the Muscular Dystrophy
Campaign on 020 7803 2856 or volunteer@muscular-dystrophy