Lobby of Parliament

We took part in a lobby of Parliament on 28 June to give people the chance to visit the House of Commons, meet their MP and explain the necessity of funding more research into neuromuscular conditions. The lobby was organised jointly by the Muscular Dystrophy Campaign, PPUK and the Duchenne Family Support Group and concentrated on the current research project into Duchenne muscular dystrophy. This is an example of the type of research which we are likely to see in the future for many other conditions and is the first test of the Government’s will and ability to fund scientists to make real progress.

Meeting with Health Minister

A delegation from the lobby attended a meeting with Andy Burnham MP a minister in the Department of Health to discuss the campaign and research funding in the future.

Nick Catlin of PPUK who attended the meeting said that the minister "fully supports the research project and is waiting for the results of the initial clinical trial before being able to commit more funding to the project."


Research into many different neuromuscular conditions is ongoing, with interesting recent breakthroughs in FSH, SMA (see www.jtsma.org.uk for more information) and Duchenne muscular dystrophy. These results are encouraging but more research is needed to help widen our knowledge of the conditions still further, and to try to convert this knowledge into real treatments or even cures.

Trials of new treatments

The Muscular Dystrophy Campaign, over the years, has put £100 million into the research which has helped to bring us this far. However, big challenges lie ahead. Laboratory research into the genetics and chemistry of the conditions is relatively inexpensive compared to the next stage, clinical trials, which begin to test treatments on real people. To get a treatment to the stage where it can be prescribed to patients is a long and expensive process. The treatment must undergo a toxicology test, to check for harmful side effects, then a number of people with the specific condition need to be recruited for a full scale trial. All this is required to ensure that new treatments are safe and effective.

After this new treatments go to a Government body, the National Institute for Clinical Effectiveness (NICE) which decides whether new treatments are effective enough and can be afforded by the NHS.
Then, and only then can new treatments be made available to all those who can benefit from them.

At any stage of the process a new treatment can fail sending researchers back to the drawing board. This could be if it is found to have harmful side effects or fails to work properly, or at all. All this means that converting the theory of how to treat neuromuscular conditions into practice and treatment will be a long and very expensive process.

We will continue to lobby MPs and Government to make them aware of the exciting theoretical research which has been done, and their responsibility to help to fund the research needed.

That is why it is important – not just to families with Duchenne but to all those with neuromuscular disorders – that this research is properly funded and can find out if the proposed treatment can be made to work and slow down or even halt the progression of the condition.