New study could lead to breakthrough treatment for those with muscular dystrophy

The “spectacular” results of a recent research project could lead to a breakthrough for those living with certain types of muscular dystrophy.

According to reports, people affected by Duchenne muscular dystrophy could be treated by a single vaccination in the near future, following a ground-breaking study led by the Royal Holloway in London.

The study assessed twelve dogs – specifically Golden Labradors – that were affected by the condition and had a life expectancy of six months or less.

Researchers at the Royal Hollaway treated the dogs as puppies and, astonishingly, found that they were all still alive two years after the initial trials.

The Labradors were treated using experimental restorative therapy, which essentially targets a faulty gene known to ‘infect’ cells with a relatively harmless virus, which later alters their genetic code. The treatment works by ‘repairing’ the cells and producing a protein thought to be essential for good muscle functioning, a report in The Telegraph explains.

Study leader, Professor George Dickson of the University of London in Egham, Surrey, said: “This is tremendously exciting progress towards a gene therapy for Duchenne muscular dystrophy.

“The studies in dogs have been spectacular and exceeded our expectations.”

Professor Darren Griffin, of the University of Kent, added: “This is really a very exciting study indeed. Duchenne muscular dystrophy is a horrible, wasting, life-ending disease of young people.

“By making use of the canine model and showing genuine improvement in the animals treated, then real hope is present for the prospect for disease treatment in humans.”

At EnViva Paediatric Care, our experienced staff can provide complex nurse-managed homecare for babies, children and young people with a range of disabling conditions, including muscular dystrophy. For more information about the services we provide, please contact us today.

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