I was 43 years of age when I found myself having to take early retirement on medical grounds due to progression of FSH Muscular Dystrophy. I had worked in Head Office of a locally based Building Society since leaving school and now faced a life relying on a wheelchair and living on benefits.

I was also single and lived alone, and became housebound until one of my sisters was able to take time from her busy work schedule to take me shopping or keep hospital appointments. As time was limited, these trips were always stressful, as I was often pushed at a breakneck speed in my manual wheelchair!

With ongoing progression of FSH, I now use an electric powered wheelchair, but as this chair is too heavy to be lifted and transported in the back of a car, I have to use public transport. However, not all public transport is fitted with a ramp, or even worse, is not in working order, which was one little problem I discovered when I found myself stranded in town one day. Thankfully, a bus inspector did come along, and contacted the depot to ensure I was able to get on the next bus, so I was grateful to get home eventually. I now tend to rely on a wheelchair taxi if I have to attend important appointments.

It was difficult to get out and socialise like anyone else, and as there was no wheelchair access into the homes of family members, I had to rely on them to come and visit me. However, as they have their own families and busy work schedules to keep to, their weekly visits were often short. With the restrictions in my life I became depressed and as time went by, I began to loose all confidence. I felt lonely and isolated, and wondered if there was anything I could do to improve my situation?

I decided to telephone Durham County Council’s Social Services Department to see if there were any options available to me, and was put through to a Social Worker called Dave. After I informed him of my situation, he arranged to visit me at home for an assessment of my care needs. Dave was friendly and helpful and the assessment went well. He asked if I’d heard of a scheme called Direct Payments. He went on to explain that, with help through Direct Payments, I would be able to employ a personal assistant who would help with shopping and some housework, and also help me get out and about to socialise. Gosh…! Help with going out to socialise..? This was just exactly the kind of help and support I needed, and the hours allocated to me would be flexible.

The next stage was a visit from Adult and Community Services for a financial assessment, and depending on my income, I was advised of the weekly amount I would contribute towards my care support in employing a personal assistant, with help through Direct Payments.

After I was happy with my contribution, I then had a phone call from Tracy who worked in the Direct Payments Office. She arranged to visit me at home where I was guided through the paperwork in employing a personal assistant. Tracy was also very friendly and helpful, and she also helped with the advert for my Personal Assistant which was then advertised in Job Centre Plus.

After a few weeks, I was delighted to meet and employ a lovely bubbly lady called Jayne. We share similar interests, as well as a sense of humour, so there’s never a dull moment! Jayne helps me with shopping, and if there’s an item that has to be reached for, or a clothes item that has to be tried on, then she is always at hand. She also helps me with some housework, such as changing the bed and cleaning windows, which are chores I can no longer do myself. On occasion, she has also helped me with some cooking, where we have “tried and tested” one or two tasty recipes!

We have also been on social outings and visited beautiful local places of interest where I have taken some photographs to remind me of my visit.

On an end note, I am so happy in the way my life has improved over these past few months, in that I am able to get out and about and socialise again, as well as having some help with household chores. Knowing I have this support in place has been a great boost in building up my confidence. I am so grateful I made that phone call that day, so thank you so much to Durham County Council’s Social Services and Direct Payments who were able to help in turning my life around. I will look back on that day in being my very own Independence Day ...!

Carol Lawson

TREAT-NMD (Translational Research in Europe for the Assessment and Treatment of Neuromuscular Disease) is an international initiative bringing together some of the world’s leading neuromuscular specialists in a pan-European “network of excellence” aimed at improving treatment and finding cures for patients with neuromuscular disorders. Linking 21 partner organisations and over 300 doctors, researchers and other professionals across 11 European countries, the EU-funded network is enabling experts to work together to share best practice and harmonise efforts on an international scale. This European network is leading the world in terms of an integrated approach for neuromuscular diseases.

Neuromuscular disorders affect over 300,000 people in Europe. The term refers to a large group of conditions that affect either the muscles themselves, or the nerves controlling the muscles. Most conditions result in chronic long term disability and early death may eventually result from respiratory or cardiac failure. TREAT-NMD will encourage experts in this field to work together to share good practice and to improve global standards of care. The network will produce treatment guidelines that describe the basic standards of care that all European patients with neuromuscular disorders can expect. At the moment, standards of care vary between different member states, thus making it difficult to conduct multi-centre clinical trials because baseline data is not consistent among these centres.

Scientists will work closely with doctors to test and apply new research into these inherited disorders, in order to develop new ways of caring for patients with conditions like Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Close links with industry partners will also be further developed in order to advance technological and methodological tools with a view to promote further clinical trials for potential new treatments and cures for rare neuromuscular diseases. Patients and patient charities will be involved at all levels of the project, which will also include a programme of training and education.

Impact of the Network

• Across Europe there is a lack of common standards for diagnosis and care of neuromuscular disorders—TREAT-NMD will propose common standards to solve this issue
• Pre-clinical evaluation of new therapies is vital and the TREAT-NMD network will ensure standardised procedures to validate relevant models and subsequent testing and evaluation protocols
• Production, toxicology and safety of potential therapeutic agents is a vital step in the clinical trials process—TREAT-NMD will ensure common standards for regulating these processes, in close collaboration with the CLINIGENE network of excellence. These standards will also be extended to ensure better targeting of systemic delivered therapeutic agents
• TREAT-NMD has created a Clinical Trials Coordination Centre that will support the translation of research into new drugs for clinical practice
• TREAT-NMD will complement other 'networks of excellence', such as MYORES, CLINIGENE, and EUROGENTEST to optimise collaborations and partnerships and to ensure that duplication of efforts is avoided
• TREAT-NMD will offer all the services needed to stimulate industry to develop new and effective treatments for neuromuscular diseases, such as pre-clinical testing and clinical trials

• Creation of harmonised, durable, and relevant biobanks and databases
• Optimising efficacy measurements of therapeutic treatments
• Dissemination of results to the scientific community
• Develop training and education programmes
• Promote knowledge transfer to industry partners
• Ensure gender equality in the scientific community
• Extension of the network to Eastern European countries

Spreading Excellence

Training and education are fundamental to the network’s goals and an online ‘clearing house’ for research placements, visiting professorships and other training opportunities for scientists and clinicians is being created with the aim of “spreading excellence” across the globe. Links with top specialists across the globe are already very strong and will ensure that everyone moves forward together on the key issues.

For more information about the network please contact the TREAT-NMD Coordinating Centre at;