The Case for Support

Muscular Dystrophy Campaign 61 Southwark Street London SE1 0HL Contact Anna Porcherot: t: 020 7803 2862 e: a.porcherot@muscular-dystrophy.org w: www.muscular-dystrophy.org Registered Charity No. 205395 and Registered Scottish Charity No. SC039445 Printed on PEFC paper, produced at a mill that is certified with the ISO14001 environmental management standard

More information on the charity can be found at www.muscular-dystrophy.org There is currently no cure for any of the 60 muscle-wasting diseases we support. Our world-class research programme funds pioneering research into effective treatments and cures, and we need your support to help fund this vital work. And to find a cure.

We are looking to raise £1.2 million to support our research into effective treatments and cures for muscular dystrophy and other neuromuscular conditions, and our community support networks throughout the UK. Background

The Muscular Dystrophy Campaign was founded in 1959 as a medical research charity that sought to discover the causes of, and find a cure for, muscular dystrophy and related neuromuscular conditions, commonly known as muscle-wasting disease.

The Muscular Dystrophy Campaign is the only national charity in the UK focusing on all forms of musclewasting disease. The scale and range of the charity’s work has grown significantly with time, and, in addition to research, we now provide a wide range of support services for those living with muscle-wasting disease.

Aims and objectives

The Muscular Dystrophy Campaign seeks to improve the lives of over 70,000 children and adults throughout the UK, affected by muscle-wasting disease, as well as the 350,000 people indirectly affected as relatives and carers. Our aim is a world where muscle-wasting disease is not a barrier to quality or length of life and does not lead to isolation and distress.

To achieve our aims, the charity has five main areas of focus: • we fund world-class pioneering research to find effective treatments and cures • we provide practical information, advice and emotional support for individuals, their carers and families 1

• we campaign to raise awareness and bring about change • we award grants towards the cost of specialist equipment, such as powered wheelchairs, to individuals and families who are in desperate need of such equipment to improve mobility, independence and quality of life • we provide specialist education and development for health professionals.

People with muscle-wasting disease, their families and carers, are at the heart of everything we do. The Muscular Dystrophy Campaign is the first port of call for information and support (emotional and practical) for those who find themselves in situations where they or a family member have been newly-diagnosed with muscle-wasting disease.

What is muscular dystrophy?

The terms ‘muscle-wasting disease’, ‘muscular dystrophy’, ‘neuromuscular conditions’ and ‘neuromuscular disorders’ all refer to a group of 60 progressive conditions that cause the muscles to weaken and waste away.

They can affect people of all ages and backgrounds, and can occur out of the blue, even if there is no family history of genetic disorders. At present, there are no cures for any of these conditions, which (depending on the type and progression of the condition) can cause severe disability and premature death.

Having a child with muscular dystrophy can sometimes feel like a constant battle.

Some doctors do not know what to suggest because it is a rare condition… I’m not sure who we would have turned to if we didn’t have the Muscular Dystrophy Campaign.

Beena Patel, mother of Ronak We’re looking for a cure...

Scientists of the future: PhD students and clinical research fellows

The Muscular Dystrophy Campaign funds world-class pioneering research into potential treatments and cures, from basic science and translational research to clinical pilot studies.

Being funded by the Muscular Dystrophy Campaign enabled me to raise other funds from other sources and helped me attend meetings where international collaborations could be set up. This ensured that there was minimum duplication of effort and faster progress.

It also allowed all of us to work with the clinicians and families. I owe a lot to the Muscular Dystrophy Campaign for lots of continued support.

Dame Professor Kay Davies

The Muscular Dystrophy Campaign’s Scientists of the Future project will offer young clinicians and scientists the opportunity to obtain specialised training in muscle-wasting disease diagnosis and management, and to be involved in ongoing research projects.

The fellowships and studentships provide clear, stable career support for talented researchers and clinicians looking to develop their careers in neuromuscular science.

In order to increase our understanding of these conditions at a basic cellular level, and to facilitate the generation of treatments, it is vital that we encourage a new generation of researchers to enter the field of muscle-wasting disease. Without such researchers in our teaching hospitals and universities, we are less likely to find effective treatment or cures for these diseases in the future.

The Muscular Dystrophy Campaign’s medical research programme has an international reputation for excellence. We invest up to £1million in such research each year, with more than 20 live projects taking place at any one time.

Among the scientists that have been contributing to muscular dystrophy research, Dame Professor Kay Davies has played a central role in trying to understand Duchenne muscular dystrophy.

www.muscular-dystrophy.org

that causes FSHD affects the ability of muscle to repair and regenerate. It will also give important information on the viability of using muscle stem cells as a treatment for FSHD in the future.

The funding that the Muscular Dystrophy Campaign provided to Kay Davies in her early years of research has been a significant factor in shaping her career, helping to facilitate her excellent work. The Muscular Dystrophy Campaign has contributed to funding her projects for more than 25 years and continues to do so today.

Dr Matthew Wood – Duchenne muscular dystrophy

PhD studentships

In line with the charity’s research strategy, the Muscular Dystrophy Campaign supports grants for PhD studentships of up to four years to encourage young scientists to enter the field of genetic research (specialising in muscle-wasting disease and related neuromuscular conditions). This is vital to ensure that there is a new generation of researchers who are entering the field of muscle-wasting disease.

The Muscular Dystrophy Campaign currently funds ten PhD studentships, and has supported more than 500 young scientists to develop careers within the field of neuromuscular science over the past 50 years.

Current PhD studentships include:

Dr Peter Zammit – Facioscapulohumeral muscular dystrophy (FSHD)

This four-year studentship, funded in partnership with King’s College London, is aimed at exploring the role that muscle stem cells have in the progression of FSHD. This research is important as it gives scientists more information on how the genetic fault

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Dr Wood, at the University of Oxford, has recently been awarded a four-year PhD studentship. The studentship is aimed at exploring ways to improve the delivery of molecular patches to the heart of those affected by Duchenne muscular dystrophy.

Clinical research fellowship

The charity has helped to establish and to fund three specialist Muscle and Nerve Centres, in London, Oxford and Newcastle. These centres provide multi-disciplinary approaches to the management and support of those affected by muscle-wasting disease, offering clinics for over 3,600 people each year; and training for clinicians, clinical trial co-ordinators and clinical research fellowships.

The training fellowships offer the opportunity for the fellows to obtain specialised training in musclewasting disease diagnosis and management, and to be involved in ongoing research projects. They will work toward a higher degree (an MD or a PhD) as part of this training.

The aim is to ensure a continuing pipeline of well-trained doctors who can understand neuromuscular conditions, and former Muscular Dystrophy Campaign-funded fellows are now the lead neuromuscular consultants in several UK hospitals.

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As with all previous fellows, it is expected that the post holder will go on to become a consultant with a specific interest in neuromuscular disorders; developing clinics in parts of the country currently devoid of such specialism and thus enhancing patient management, which is a major objective of the Muscular Dystrophy Campaign.

Dr Reghan Foley is one of three Muscular Dystrophy Campaignfunded clinical research fellows. She is currently working on congenital muscular dystrophies at the Dubowitz Neuromuscular Centre at Great Ormond Street Hospital in London.

Funding from the Muscular Dystrophy Campaign has made possible my current clinical research fellowship and research degree studies and has provided me with essential protected research time. This is a tremendous opportunity both for my training as an academic clinical researcher in neuromuscular diseases as well as for my research in the congenital muscular dystrophies. The timing of this opportunity has corresponded with the emergence of potential therapies in the congenital muscular dystrophies (CMDs). I feel privileged to be a part of the efforts of helping the CMD patient population progress toward clinical trial readiness at this exciting time.

Dr Reghan Foley

Supporting families...

Network MD Muscle Groups

Throughout the UK, regional community support networks have been established to bring together individuals and families living with muscle-wasting disease, offering them the chance to meet others in similar situations, share knowledge and offer support.

The Muscle Groups meet quarterly and provide families with vital peerto-peer advice, support, advocacy and information, which they might otherwise find difficult to access.

As well as sharing information on condition management issues (with workshops provided by attending clinicians, all experts in their field), the groups are also encouraged to get involved with local campaigning and fundraising activities.

The physical effects of muscle disease can often lead to emotional difficulties, such as depression and withdrawal. The peer-to-peer support on offer has proved to be an invaluable tool for improving the quality of life of those living with muscle-wasting disease, reducing feelings of isolation and empowering individuals to engage with others who have experienced similar challenges.

Families, clinicians, health professionals, NHS decision-makers and parliamentarians come together in these groups, where they have created a shared vision, increased volunteer participation and directly influenced the NHS decision-making process.

I left the meeting feeling that finally, after years of feeling abandoned, something is being done to help me. I feel positive and look forward to the next meeting.

Valerie Wintle, Muscle Group member www.muscular-dystrophy.org

Trailblazers – young campaigners network

The Trailblazers project is a nationwide network of campaigners who fight social injustices experienced by young disabled people. Founded in early 2008 as a mentoring, support and campaigning network for young people aged 16 to 30, Trailblazers is open to all young disabled

Costs

people and not just those living with muscle-wasting disease. As well as discussing national disability issues and exchanging knowledge, the members of Trailblazers develop local networks to meet and make friends with other young disabled people who have to deal with similar situations.

We are currently seeking funding to ensure that the Muscular Dystrophy Campaign can continue to run these two projects.

We have applied and will continue to apply to a range of suitable funding sources throughout the UK, including trusts, corporate supporters and individual supporters.

Being part of the Trailblazers project also offers young disabled people the opportunity to develop new skills such as writing, design, event planning and public speaking. It also encourages them to work in conjunction with the experienced campaigners from the Muscular Dystrophy Campaign, offering valuable work experience to a group who often struggle to find employment.

The first network of its kind in the UK, almost every aspect of the Trailblazers is driven by its volunteers, all of whom are young disabled people. The project as a whole is made up of four distinct elements: networking – volunteers attend regular regional meetings across the UK, giving them the chance to meet and connect with other young people in similar situations training – with professional training, work experience and participation in the campaigning activities of the network, volunteers are encouraged to develop new skills in the fields of writing, design and planning investigating – since it started, the Trailblazers project has published five investigative reports following surveys into disability access and inclusion in public transport, leisure facilities, higher education, tourism and employment opportunities, all of which have received widespread national media coverage raising awareness – the network has been consulted by numerous decision-makers seeking advice (from MPs, government departments and NHS trusts, to multi-national corporations, key stakeholders and other charities) and has also been instrumental in securing the formation of an All Party Parliamentary Group for Young Disabled People.

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Total projects costs over four years: Scientists of the future

Network MD

3 x PhD studentships

Clinical research fellowship

Muscle Groups

Trailblazers

Total over four years

Year 1

106,440

58,300

56,260

79,000

300,000

Year 2

106,440

58,300

56,260

79,000

300,000

Year 3

106,440

58,300

56,260

79,000

300,000

Year 4

106,440

58,300

56,260

79,000

300,000

Total project costs

425,760

233,200

225,040

316,000

1,200,000

Help us to raise £1.2 million by:

• making a four-year pledge • making a one-off donation • committing to a regular monthly donation • introducing us to a prospective individual, grant making trust or business donor • joining our network of campaign supporters and volunteer fundraising

champions • recommending us as your Charity of the Year.

Scale of giving

The chart below gives an illustration of how, with your support, we intend to reach our target so we can continue to run these projects: Annual donation

Gift over four years

Number of pledges required

Total over four years

250,000

n/a

1

250,000

50,000

200,000

1

200,000

25,000

100,000

2

200,000

15,000

60,000

3

180,000

10,000

40,000

4

160,000

5,000

20,000

6

120,000

2,500

10,000

9

90,000

Total

1,200,000

Making the most of your gift - Gift Aid it!

The Muscular Dystrophy Campaign can claim Gift Aid from the Inland Revenue on all eligible donations made by individuals.

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Researching for cures...