By Dave Gudgeon

Last updated at 18:12, Friday, 24 October 2008

Brave schoolboy William Tweddle today begins a pioneering course of treatment that could extend his life and those of other children.

William Tweddle with his mother Jodie and her partner Gary Birkett

The seven-year-old suffers from the rare muscle wasting disease Duchenne Muscular Dystrophy. It means he is likely to end up in a wheelchair and may die prematurely.

Now he is one of 150 youngsters across the world, chosen to take part in clinical trials for a drug developed by an American company.

His mum Jodie, of Randall Street, Denton Holme, Carlisle, is hoping the drug – PTC124 – will extend his life and improve his abilities.

She said: “Duchenne is the most acute form of muscular dystrophy and the aim of this new drug is to transform it into what is known as the Becker type of muscular dystrophy.

“If PTC124 works then it will help Will and other youngsters who are diagnosed with Duchenne in the future.

“It is a very exciting prospect and shows that research is paying off.

“When we got a call from the staff at the Newcastle hospital where William receives regular treatment to offer us the chance to take part in these clinical trials, we had no hesitation in saying yes.”

William travelled to Newcastle with his mum and her long-term partner, Gary Birkett, yesterday for tests and will begin taking the drug later today.

Jodie said: “Will has to take the new drug three times each day and travel to Newcastle every six weeks for two days of tests to ensure that his body is not rejecting the treatment.

“We also have to keep a diary recording when he takes the drug and any abnormal reactions. The clinical trial lasts for a full year and if there are no problems Will automatically keeps taking the drug.”

Gary recently raised £2,000 by climbing Ben Nevis, Scafell Pike and Snowdon with friend Marco Hartley, a lab manager with Specsavers Carlisle.

The pair managed to complete the mountain marathon in 22 hours 55 minutes, despite having to climb Lakeland’s highest peak in darkness.

Jodie said: “It just shows that the money raised by people like Gary and Marco is having a positive impact by funding research into things like PTC124.

“After their efforts were featured in the paper I heard from a teacher at Brampton’s William Howard school who wants to donate the takings from a show there to the cause. That’s fantastic.”

William, who has just started at Caldew Lea school, is one of just a handful of sufferers of Duchenne in Cumbria and is believed to be the only one taking part in the trials.

He is given 20 minutes of physio every day by his mum, faces a lifetime on steroids and already goes for check-ups several times a year.

Jodie added: “He’s a very bright and articulate boy we know in future he is likely to suffer behavioural as well as physical problems. But this treatment might make things better than expected.”

First published at 05:16, Friday, 24 October 2008
Published by http://www.cumberlandnews.co.uk