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07 October 2019

We’re helping tackle a rare brain disease that affects children

By Dr Susan Campbell and Dr Elizabeth Allen | Twitter

Monday 9 December • Reading time: 2 minutes

Vanishing White Matter (VWM) is a rare neurodegenerative disease that largely affects children. Sheffield Hallam researchers are helping to tackle this currently incurable illness.

Vanishing White Matter (VWM) is a rare neurodegenerative disease that largely affects children.

It causes children’s brain cells to become damaged and depleted, with devastating effects. Typically, children lose the use of their muscles – and have a life expectancy of 5 to 10 years. There is currently no cure.

A team of bioscientists here at Sheffield Hallam have received funding from Great Ormond Street Hospital Charity and Sparks to learn more about the disease, in the hope of helping the children and families affected.

One of the problems with Vanishing White Matter disease is that it’s so hard to diagnose.

Our scientists are working on a new technique to aid the diagnosis of VWM. This involves reprogramming children’s skin cells so that they act like brain cells. It means doctors could use these skin cells to diagnose the condition and work out the best treatment.

As skin cells can be retrieved simply with a needle, this technology would make diagnosing VWM much easier and quicker, giving the child more chance of getting the specialist clinical care they need.

What’s more, the wider research could be useful in other, more common neurodegenerative diseases such as Alzheimer's and Parkinson’s – all diseases which, like VWM, are based on brain cells not being able to make proteins properly.

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