A researcher from Britain’s esteemed Oxford University has said that a new treatment for men and boys with Duchenne muscular dystrophy (MD) could be available in the UK within five years or less.
Professor Kay Davies announced that trials of a new drug geared at maintaining patients’ muscles through doses of utrophin were scheduled to begin before the close of 2017.
Speaking at the Oxfordshire Science Festival, she said: “Duchenne is horrible for these boys. They normally get diagnosed at the age of four of five, and they suffer from a progressive muscle wastage which leaves them in a wheelchair by the age of 12.
“Somewhere in the next five years, we will be able to do something for these boys, to stop them from going into a wheelchair, and perhaps prolong their life and improve their quality of life.”
It is estimated that the rare muscle-wasting condition affects approximately 2,500 boys and men in the UK.
However, recent trials using mice and other small animals have discovered that increasing the levels of utrophin in the body could compensate for a similar protein, dsytrophin, which is typically sparse in male MD patients.
“It [MD] affects every population in the world and we need to have a treatment in pill form. This is why I am so keen on our approach, because this is something anyone can take with their breakfast or dinner,” said Professor Davies.
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