We are delighted to announce that the Myotubular Trust have been awarded a substantial grant funding award and have been guaranteed a minimum of £25,000 in 2008 to pay for setting up a new Patient Registry.

The Patient Registry will be a database of very basic patient information which will allow, when the time comes, efficient, fast track, local, clinical trials of new treatments in Europe. Dr Heinz Jungbluth, Senior Lecturer and Consultant in Paediatric Paediatric Neurologist in charge of the Neuromuscular Service at the Evelina Children's Hospital, Guy's and St Thomas' NHS Foundation Trust, London has been appointed as a co-ordinator and will liaise with other leading European academics in this field to ensure the robustness of the patient registry.

With part of the grant money, the charity are also enlisting the expertise of a nurse administrator whose job will be to liaise with patients and their clinicians to collect and input the data, so that the information is consistent. The charity will also establish an ethical steering group to control release of data to interested researchers.

The registry will be put together by the Myotubular Trust working in partnership with TREAT-NMD (Translational Research in Europe - Assessment and Treatment of Neuromuscular Diseases), a European neuromuscular network addressing the fragmentation currently hindering translational research for cutting edge therapies in rare neuromuscular diseases (NMD).

It is envisaged that the registry will take the form of an electronic database that will:

• Encourage development of more research.
• Provide neuromuscular researchers with specific patient information to assist their research.
• Help with new clinical trials in Europe.
• Enhance clinicians' and other health professionals' ability to efficiently deliver treatments for this disease.

The database will be built to be compatible with a generic database that has been developed for other more common neuromuscular disorders.

Jeans for Genes is a national charity which raises money to fund care and support for children and their families affected by genetic disorders. It also funds research into the causes and cures of these disorders. The Myotubular Trust is one of eighteen charities which will benefit from the proceeds of this year's Jeans for Genes Day, which takes place on Friday 3rd October. The Day invites everyone to go to school or work in their jeans in return for a donation. The Myotubular Trust was specifically chosen as the most deserving charity for the award, and had to undergo a selection process culminating in a presentation to the Jeans for Genes Chief Executive Officer and Board of Trustees.

Not only will the grant money make a huge difference to research and treatments for affected children and adults, but the Trust's partnership with Jeans for Genes will also raise the profile of the condition and the Myotubular Trust, within the wider public, giving the charity the opportunity to become a familiar name. Statistics show that 80% of people will only give to a charity that they have heard of before, so becoming more widely recognised could make all the difference between someone choosing to donate or not in the future.

So, now the Myotubular Trust really needs your help in three areas, to make this Jeans for Genes initiative as successful as it can be.

• If you live in the UK, please encourage your school, college, university, social group or place of work to support 'Jeans for Genes'. Perhaps ask your friends and family to get their organisations involved too. Visit the Jeans for Genes website to request an information pack.
• Jeans for Genes have asked for UK case studies to help gain publicity and funding for the campaign and raise awareness for the charity, through local media. You need to give your permission to be contacted but after that, you won't need to do anything other than tell a newspaper, radio station, or tv broadcasting company how you or your child or relative has been affected by the condition, and the Trust can even provide you with a standard press release, so that you could provide a standard charity message about the Jeans for Genes campaign. This case study may also be used on the Jeans for Genes website or in Jeans for Genes school packs.
• Jeans for Genes have also asked if any UK families, their friends or family members are willing to spare a little time to speak on a voluntary basis in their local school assemblies about the condition around the time of the event. The Trust can provide you with a full script / guidelines and support materials for this, all you would need to add would be your personal experiences. Jeans for Genes are also offering theopportunity for you to attend a course led by a trainer in public speaking if you so wish but again, your consent and contact details are required. If you would like to volunteer to be a school speaker, please download the registration form and send this to Jeans for Genes with a copy to Wendy at the Myotubular Trust.

As a rare condition, we all need to work together to bring about finding a treatment or cure. And the more families that are willing to help achieve this, through the Jeans for Genes initiative, the more chance we have to fully maximise awareness for the myotubular and centronuclear myopathies. The Myotubular Trust truly hopes all UK families will give this wonderful opportunity their full backing and support in 2008.

For further infomation about the grant, visit the Jeans for Genes website
or to take part, please contact Wendy at the Myotubular Trust.

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