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Dame Kay Davies, Dr Lee’s Professor of Anatomy here in DPAG, has been working on a clinical trial that today announced a potentially life-changing discovery.

Summit Therapeutics, the drug discovery and development company advancing therapies for rare diseases and infectious diseases, for which Professor Davies is a co-founder, have released the interim results from the trial, PhaseOut DMD, which had been evaluating the effect of ezutromid in patients with Duchenne muscular dystrophy, DMD.

Discoveries show that ezutromid does indeed reduce muscle damage significantly in DMD patients; a total of 14 out of 22 patients showed a decrease in developmental myosin, a biomarker of muscle damage, with five of those patients showing a greater than 40% reduction.

DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The results from the trial suggest a potential universal treatment for DMD, since there is currently no cure for DMD and life expectancy is into the late twenties.

Professor Davies is delighted at the results from the trial, saying: These data provide the first evidence of utrophin modulation working in patients. If further findings build on this evidence they could establish ezutromid as a universal, disease-modifying treatment and bring hope to all patients and families living with DMD.

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