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Tuesday 23rd January 2018

Gene trial for muscular dystrophy

22nd October 2007

A gene therapy trial is starting in London to try to extend the lives of patients who suffer from the fatal disorder Duchenne muscular dystrophy (DMD).

A small group of patients will be injected with an experimental drug that will target the faulty gene that causes the progressive muscle wasting of the condition in what is the world’s first trial of this type. The lead researcher Professor Francesco Muntoni of Imperial College London stresses the trial may not provide a complete cure but will buy time for children with the condition.


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