making muscle wasting history

I attended the Lobby of the Scottish parliament today 3rd June 2009. 60 families and young people attended the day and met with MSP's. Health Minister Shona Robison and First Minister Alex Salmond attended the meeting after the lobbying in the assembly building.

We put our argument forward to urgently establish a centre of excellence for Duchenne in Scotland. All families in Scotland are supporting this demand. The evidence is very clear from the publication of Treat NMD's standards of care that only expert duchenne clinicians and health professionals working as a team in one place can deliver the essential medical care our sons need.

But we also have the prospect of delivering personalised medicine regimes for individual patients using steroids and heart drugs in combinations with new drugs like PTC 124 and AVI morpholinos now in clinical trials. These medicines could significantly slow the progress of the severe muslce wasting while we look for the magic bullet drug to cure duchenne. But this will significantly increase the demands on clinical teams to assess, support and monitor the medical progress of our children and adults living with Duchenne. We need expert centres like Great Ormond Street and Newcastle that can deliver these services. We also need more centres delivering clinical trials for Duchenne drugs.

It was a real shame that Shona the health minister did not take this argument on board. Rather she has agreed with the Muscular Dystrophy Campaign and the Scottish Muscle Network (Clinicians) to undertake a review of neuromuscular services in Scotland. She agreed this early this year and it has still not even started.

We need some clear and bold political leadership here to say the evidence is already there for Duchenne and we will now set up a Centre of excellence for Duchenne in say Edinburgh and tie it into the excellent University medical research centres in the city. They need to say that they will make this an international Centre of excellence to provide one of worlds leading clinical and research facilities for Duchenne. They will network with Newcastle and Great Ormond Street to provide an unparalleled basis for trialling new drugs and therapies.

Dr Karen Nasmith from the Scottish Muscle network spoke at the meeting about their plans to expand the network in Scotland with teleconferencing offering a way for Duchenne patients to access clinical and medical expertise. But the reality of this loose network approach will only to be to continue with the postcode lottery. Some patients will get parts of the medical care package while others will not. This is the current experience of patients in Scotland after 10 years of the muscle network.

We need Duchenne expertise in one place so that teams of neuromuscular consultants, physios, cardiologists, respiratory consultants, endocrinologists and others get to know in detail how duchenne is affecting our children and men with Duchenne. That personalised medicine regimes can be delivered and long term effects of drug regimes can be properly monitored. Loose muscle networks and virtual conferencing will not achieve this for Duchenne.

Some families travelled for over 4 hours to get to the lobby and told the meeting that they would be happy to travel that far to a Centre in Scotland. Geography is not the issue. We really do not need reviews that last years for Duchenne. The evidence for a Centre in Scotland is already overwhelming.

A brilliant meeting with many families expressing their concerns and anger about poor provision. Provision for adults with Duchenne is very poor. So we will have to keep up the pressure to get rid of the postcode lottery of medical care in Scotland. A big thank you to everyone who came and to Dean Widd for organising such an excellent event.