Campaign Spring 2012

Campaign The newsletter for our supporters

Spring 2012

Meet our inspiring supporters

www.muscular-dystrophy.org

020 7803 4800

Welcome to your first issue of Campaign for 2012. Inside you’ll find lots of exciting news about the charity’s work which you have helped support, and learn more about our research grants, current campaigns, fundraising ideas and much more.

In particular you may like to read our article on page four that features the charity’s advocacy work to date. This is an initiative that as you can see is helping more people and families living with muscular dystrophy and related conditions.

It is only with your help that we can achieve such great success and help more people to receive the support and benefits they deserve every day.

I was delighted to meet Lynn and Paul Leadbitter at our National Conference in October last year, and to hear about their dedication to fighting for – and then winning – their son, Matthew’s, care and support. It was an honour for me to meet so many of the charity’s supporters at the conference, which you can read about on page three.

On the following pages you’ll read more about the research grants we have funded, including a new project into exon skipping and delivery of molecular patches for the heart for Duchenne muscular dystrophy.

Finally I’d like to highlight the many wonderful fundraising events and activities that our supporters are organising and thank them, and you, for your continued support. This support is vital to the charity; it is what makes supporting families living with muscular dystrophy and related conditions possible.

Thank you.

Sue Barker MBE President, Muscular Dystrophy Campaign Thanks to your valued support, we are a major UK funder of research into muscular dystrophy and related conditions. Each year we invest around £1m into research that we hope will lead us closer to developing effective treatments.

To keep this vital research going, we chose to make today count in 2012. We invited people across the country to take advantage of the extra day in the leap year to take part in one of 29 February – make today count our exciting fundraising events.

Our own Research Director, Dr Marita Pohlschmidt, joined 99 others and sky dived for us; we had over a dozen good old-fashioned bucket collections too and many companies donated the extra day to raffle as a day’s leave for one lucky employee.

Thank you to all our generous supporters for your time and dedication.

To support us call 020 7803 4834

News

Conference round-up During October last year more than 400 people joined us at the Scottish and National Conferences in Glasgow and Nottingham.

Our conferences are a great opportunity to talk about our work, and to honour the outstanding contribution of supporters with our awards. We were delighted to have our President Sue Barker in attendance to present the national awards.

Among those honoured were Chloe Ball Hopkins (pictured on our cover), who won the Young Inspiring Person of the Year President’s Award for her bravery in appearing live in front of 375 million viewers when she performed the coin toss at the Wimbledon Ladies’ Final in 2011.

The late Reg Mackie was also honoured in Scotland with our Lifetime Commitment Kite Award.

Reg campaigned and fundraised for the charity for more than 50 years.

The research sessions were popular with families, as were the sessions on working with the media and our Trailblazers. In a Muscular Dystrophy Campaign first, the National Conference was streamed online, making it possible to follow the event live on the web.

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The 2012 conference dates are now set. Scottish Conference: Saturday 6 October 2012, at the Beardmore Conference Centre in Glasgow. National Conference: Saturday 13 October 2012, at the East Midlands Conference Centre in Nottingham.

Christmas cheer Thank you to everyone who supported us last Christmas through our festive fundraising.

Our online Christmas shop went down a treat and together you helped us raise a staggering £30,000 simply by buying our Christmas gifts and products.

Exclusive cards by Christmas card competition winners Liz and Alison sold extremely well and our new design competition for 2012 will open soon. In our Christmas appeal we featured nine-year old Pascal Bailly, who has Duchenne muscular dystrophy, and we shared some of his Christmas wishes.

Thanks to your generous support, we received not only more than £20,000, but Pascal also received his wished-for Lego model.

Christmas revellers raised their voices at our Spirit of Christmas carol concerts across the UK in November and December, from Paisley in Scotland to Winchester in Hampshire.

The jewel in the Christmas crown is traditionally the carol service held at Oxford’s Christ Church Cathedral and last year’s event lived up to its reputation. Oxford local, Tracy McMahon, who is a manifesting carrier of Duchenne muscular dystrophy, lit the first candle on the night.

“It has got to be one of the best evenings of my life, the readers were very funny and amazing to listen to in such beautiful surroundings – it felt a very special evening for all,” Tracy said.

www.muscular-dystrophy.org

Campaigning

Fighting for people’s Lynn and Paul Leadbitter won our 2011 President’s Award for Carers of the Year, at our National Conference.

As carers for their son, Matthew, who has Duchenne muscular dystrophy, they were locked in a battle with their local NHS to secure funding so Matthew could receive care at home, following his recent tracheotomy, rather than in a care home. Our advocacy service took on this case for the family and won; funding was agreed for adaptations that made it possible for Matthew to go home at the end of last year, just in time to spend Christmas with his family. “It’s wonderful to see Matthew laughing and joking; he seems a different lad and chirpy, his old self,” Lynn said.

Our advocacy service was officially launched in April last year to offer expert advice and support. More recently, the team has the benefit of legal representation thanks to the generous support of an extensive team of legal

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Lynn and Paul Leadbitter with Sue Barker

experts at law firm, Hogan Lovells.

We developed the service in response to an increasing number of individuals and families needing our help in getting the necessary care and support to live full and independent lives.

Our advocacy service has continued doing exactly that and throughout 2011,

we secured more than £300,000 in terms of welfare benefits, care packages and adaptations which had previously been denied by authorities.

For example four-year old Joshua Twine, who has Duchenne muscular dystrophy: when his mother, Charmaine, got locked in a battle with Southampton

If you’re struggling to access the benefits and services you’re entitled to, do get in touch with David Moore-Crouch in our advocacy team on 020 7803 4808 or email campaigns@muscular-dystrophy.org We are here to help you.

To support us call 020 7803 4834

Campaigning

rights to services Council who refused to offer any support for Josh’s transition into year one at school, our advocacy team stepped in to help Charmaine fight the council’s decision.

“I’m so glad I never gave up and I had the Muscular Dystrophy Campaign by my side, who listened to what I had to say and offered supportive evidence that made all the difference.”

Our team worked with the Local Education Authority and Josh’s school to get the necessary support in place,

and within three months, Charmaine got the news she was hoping for: Southampton Council reversed their decision and put in place the necessary support to meet Josh’s health needs while enabling him to reach his academic potential and fully integrate into the school community.

Charmaine’s fight is not a rare one. But Josh’s story highlights what can be achieved when families keep fighting and take advantage of the support that is available.

Josh Twine – another advocacy success

National recognition for our campaigners Campaigners in our Muscle Groups were acknowledged for their hard work and dedication, when the Muscular Dystrophy Campaign won ‘Campaigning Team of the Year’ at the 2011 Charity Times Awards. The panel recognised the dramatic increase in care advisors in post across the UK rising from only 13 in 2010 to the current total of 31.

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Our campaigns team

Join our dedicated and determined campaigners around the country and add weight to our fights for the rights of thousands of families with neuromuscular conditions to have access to specialist support, as well as vital emotional and practical advice.

If you’d like to get involved, do get in touch with Lisa James in our campaigns team on 020 7803 4865 or email campaigns@muscular-dystrophy.org

www.muscular-dystrophy.org

Research update

Research priorities Research is, and has always been, the Muscular Dystrophy Campaign’s top priority. Over the past 50 years, we have funded world-class research that has not only been groundbreaking, but has also laid the foundation for further discoveries which may lead to treatments and cures. All of this pioneering work is possible only through the kind support and generous gifts from you, our supporters.

Each year we provide funding to between 25 and 30 research projects and this is so important in bringing hope to our families. Here are four examples of new research projects we have recently awarded funding to.

Understanding limb girdle muscular dystrophy Professor Kate Bushby at Newcastle University is supervising a project to continue her work on the limb girdle muscular dystrophies (LGMD) with the aim of developing an effective treatment for people living with the conditions.

There are at least 21 different types of LGMD. The precise mechanisms underlying two of these – LGMD 2B and 2L – are currently unclear, making it harder to design therapies. It is thought that the genes causing these conditions are both involved in muscle repair after injury. This project aims to understand how these genes function and relate to each other, which will bring a new level of understanding of LGMD2B and LGMD2L. This could result in new therapeutic approaches to improve muscle repair and thereby treat these types of muscular dystrophy.

To support us call 020 7803 4834

Testing therapies Dr Susan Brown at the Royal Veterinary College will supervise a student to study two conditions caused by changes in a gene called FKRP. It is not well understood why a change to one part of the FKRP gene can cause a very severe form of congenital muscular dystrophy while a change in a different place can cause a milder form of limb girdle muscular dystrophy (type 2I).

They will use an animal model to try to understand these conditions better and identify where it would be most effective to intervene with a therapy. They also plan to investigate the viability of possible therapeutic approaches such as increasing the amount of a protein called LARGE which may be able to compensate for the changes to FKRP.

Research update

Improving delivery of molecular patches to the heart Professor Dominic Wells at the Royal Veterinary College will supervise this project to improve exon skipping technology for Duchenne muscular dystrophy. Exon skipping is currently in clinical trial as a potential treatment for the condition. It is proving to be a challenge for sufficient amounts of the molecular patches to travel from the blood stream where they are injected, into all the muscles of the body and particularly the heart.

This project aims to investigate ways of making the blood Professor Francesco Muntoni vessels more ‘leaky’ so that more molecular patch is able to at University College London pass from the blood stream into muscles. Improving the will supervise this project delivery of molecular patches has the potential to vastly aiming to improve the improve the effectiveness of exon skipping and may allow diagnosis of congenital the use of lower doses, which will be safer to use and muscular dystrophies and cheaper to produce. myopathies. The congenital muscular dystrophies and myopathies are a diverse group of conditions and The last issue of Campaign reported that the University of numerous gene changes have Oxford’s Professor Kay Davies was calling for the already been found to cause implementation of newborn screening for Duchenne muscular them. However, there are still dystrophy to ensure early diagnosis. Wales used to be the only many people for whom a country in the UK where newborn screening for Duchenne precise genetic diagnosis muscular dystrophy was carried out. But at the end of 2011 the cannot be given as they do not test was stopped because it was regarded as unreliable and have a change in one of the one in five boys with Duchenne muscular dystrophy was missed known genes. in the diagnoses.

This project aims to use In December, members of our Welsh Muscle Group including cutting-edge technology – Jeanette George, whose son has Duchenne muscular dystrophy, whole genome sequencing – took their concerns to Cardiff and Vale University Health Board, to search for new causative telling of the invaluable role of the newborn screening service. genes. Being able to give a With help from our supporters, the Muscular Dystrophy precise genetic diagnosis will Campaign is working closely with researchers and clinicians to allow families to have genetic develop a safer test. We will lead the campaign to make any counselling and a more new test part of a newborn screening programme that will be accurate prediction of how available for the whole of the UK. their condition will progress.

Identifying new genes

Newborn screening fight continues

www.muscular-dystrophy.org

Real story

A sports-loving man of courage In true sibling fashion, Peter Wood referred to his late brother David both as ‘Mr Belligerent’ but also as ‘the most courageous person I’ve ever known’. “If I had an ounce of his stamina and belligerence, I’d be doing really well. He really had loads of courage,” Peter said.

Peter got in touch with the Muscular Dystrophy Campaign last year, soon after his younger brother, David (50), lost his battle with a severe heart disease. Peter and his wife, Jane, were organising David’s funeral and asked their friends and family to donate to the Muscular Dystrophy Campaign in lieu of sending flowers. David had Becker muscular dystrophy.

“People were really surprised when they saw us wearing the orange Muscular Dystrophy Campaign T-shirts to David’s funeral, but they soon understood the

significance of the gesture,” Peter said.

“Many of the guests said they wished they’d had T-shirts too, as they felt it was a fitting tribute to my brother.”

David was born and grew up in Hornchurch in Essex. As a young boy, he had difficulty walking and the doctors told his family he was ‘a bit pigeontoed’. As a young teenager, it was clear that David was dealing with something more serious and he was diagnosed with muscular dystrophy. His family was given little information or support, and it was only when David was about 30, and in a wheelchair, that he discovered he had Becker muscular dystrophy.

A creative and artistic man, David kept himself busy with design work as long as he could. A huge sports fan, he loved watching cricket and was described by his older brother as ‘the oracle’, who

David Wood

knew ‘everything there was to know about cricket’.

In 2010, David contracted double pneumonia and septicaemia. He was on lifesupport and the medical team didn’t think he would pull through. In ‘true belligerent character’, he did.

Two weeks after his 50th birthday in July 2011, David sadly died. Peter, who has inherited David’s cat, Mason, said he had fond memories of his brother and that by talking about the ‘man who always had a smile on his face and a cheeky sense of humour’, he is keeping David’s memory alive.

Muscular Dystrophy Campaign is grateful to Peter and Jane Wood and their family. ✱ The Their generous support at their time of loss makes such a positive difference to the vital work of the charity. If you’d like to remember a loved one in this way, please contact Sarah Greenwood on 020 7803 4834 or s.greenwood@muscular-dystrophy.org

To support us call 020 7803 4834

Events

Running in orange Hole in one!

We had a fabulous team of dedicated and enthusiastic runners take part in the Great North Run last year.

More than 120 people, dressed in their ‘stand out from the crowd’ orange T-shirts and took to the streets of Newcastle and South Shields. Together they raised over £70,000 towards our vital work.

Jed Thirkettle, 11, (pictured) took part in the junior run the day before, with his friend Tom Petrie. Jed did incredibly well and completed the 4km run – not bad when you have Ullrich congenital muscular dystrophy – and that in turn inspired his dad, Michael, to complete the race the next day. ‘Team Jed’ was set up in honour of Jed and his team of 31 runners – including his headmaster and teachers – ran in the 31st Great North Run. If you’d like to run in orange for us in 2012, we have plenty of runs to choose from, so do get in touch.

Just visit www.muscular-dystrophy.org/running

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Forest fundraiser

Golf raises £40,000

Last year’s Four Course Classic golf challenge, where players were sponsored to complete four 18hole golf courses in one day, raised over £40,000 for the charity.

Henry Jacob, whose team won the top fundraising prize, said, “Taking part in the golf marathon was one of the most rewarding experiences of my life. Yes it was painful and yes it was a lot tougher than we thought, but I would recommend it to anyone. This truly is a great day to be a part of.”

This year’s event already looks just as promising, with some great celebrity support from star player Nick Faldo who has donated a fantastic signed prize. take part in this year’s ✱ Toevent, on Friday 22 June, please contact Hayley Gill on 020 7803 4824 or email h.gill@muscular-dystrophy.org

Dedicated supporters Emma Hallam and her husband, Andy, started fundraising for us after their four-year-old son, Alex, was diagnosed with Duchenne muscular dystrophy in April 2010. Since then, Andy has taken part in the London Marathon along with four friends, while Emma has organised a successful golf day which took place in September.

They have now formed a volunteer fundraising committee and hope to continue raising funds by, among other things, organising their biggest event yet – signing up 300 participants to take part in a 20-mile cycle ride in Charnwood Forest in Leicestershire on 29 April.

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If you would like to help them in their fundraising efforts please contact Charlotte Allen on 0121 779 4488 or visit www.charnwoodforestcycleride.co.uk

www.muscular-dystrophy.org

My story

Boccia for all Boccia is really a sport for everyone says Scott McCowan, Peter McGuire, Stephen McGuire and Jacob Thomas who are professional, elite athletes who play the sport for Great Britain. All have muscular dystrophy and all are likely to represent ParalympicsGB in Boccia in London later this year.

Jacob, from Pembrokeshire in Wales, has Duchenne muscular dystrophy and was introduced to Boccia in primary school. In the last two years, Jacob has become Welsh Champion 2010 and British BC3 Champion 2010 and 2011. He has always loved sports of all kinds, but finds Boccia gives him a chance to compete on equal terms.

Scott, from Ayrshire in Scotland, also has Duchenne muscular dystrophy. He initially thought Boccia was something he wouldn’t be able to play and regrets missing out on a few good years before starting to play Boccia four years ago with his dad as ramp assistant. A natural, Scott counts captaining his country in the 2010 World Championships and beating the World number 2 pair in 2011 among his proudest moments in

sport. He is ranked world number 25.

Peter and Stephen grew up in Hamilton in Scotland and both have an undiagnosed form of muscular dystrophy.

Stephen has always loved sport and was determined to find a Paralympic sport that he could compete in. After playing Boccia at a taster event, he was invited to the European Championships in Portugal and soon enlisted Pete to partner him in the pairs. They have gone on to achieve world number two ranking as well as gold medals at the 2009 European Championships and silver

10 To support us call 020 7803 4834

Jacob Thomas

medals at the 2010 World Championships.

Individually, Pete – who describes Boccia as ‘like a game of chess’ – is ranked ten in the world and 2008 Scottish and British Champion, while Stephen is world number two and gold medallist at the 2009 Czech Open, silver medallist in the 2010 World Championships, three time Scottish and Scottish Open Champion and six time British Champion.

“With hard work and dedication to the training required, you can get to a high level in this sport very quickly,” Stephen said.

Boccia (pronounced Bot’cha) is a highly competitive, Paralympic sport for males and females with impaired functional ability in all four limbs. Players are divided into four classifications, depending on their disability and functional ability. Boccia is a target ball sport, belonging to the same family as pétanque and bowls, and is played indoors on a court similar in size to a badminton court. Boccia has no Olympic counterpart.

News

Trailblazers target hate crime Young disabled people are experiencing an alarming number of situations where they face disability hate crime, says a Trailblazer report released in Febuary.

The investigation and report were prompted by Trailblazers speaking up about being humiliated, harassed and bullied by non-disabled people.

The network of young disabled campaigners received more than 100 responses to their survey and found that shockingly: l eight out of ten young disabled people say they have been harassed, humiliated, or embarrassed by a person’s attitude to them being a disabled person l 50 percent of respondents say they have been intimidated because they are disabled.

Talking about her experiences, Trailblazer Jagdeep Sehmbi from Birmingham said, “Whilst waiting at the bus stop, two girls were laughing and mocking the contractures I have in my wrists.“ Sadly, experiences like this are all too common and the Trailblazers are calling for the police, the government and other relevant authorities to make positive changes, from setting up a well-publicised helpline number for people to call and report incidents of disability hate crime, to organising a national awareness campaign that helps define disability hate crime. more information about the report or to get ✱For involved with the Trailblazers visit www.musclar-dystrophy.org/trailblazers

Jagdeep Sehmbi stands up to hate crime

Diving with danger Gary Bennett and Dan Bacon took a dive with danger raising more than £2,100 sponsorship from family and friends by diving with sharks last November.

Setting themselves the challenge to raise funds for the Muscular Dystrophy Campaign, led Gary’s wife Sarah to sign up for a sponsored sky dive this year.

Gary’s daughter Abbi, has Ullrich congenital muscular dystrophy, and Gary and Sarah and their family and friends are determined to raise as much money as they can to help fund research into Ullrich and other muscular dystrophies.

www.muscular-dystrophy.org 11

Our information is top quality Many of the 70,000 people affected by muscular dystrophy and related conditions rely on us to provide up-to-date and accurate expert information – as well as advice and support. We are pleased to announce that the quality of the information service we provide has now been given national recognition with the recent Award of the Information Standard.

A quality mark created by the Department of Health that helps people to choose reliable health and social care, the Information Standard was awarded following a rigorous review and accreditation process. need information about muscular dystrophy âœą Iforyou related conditions then call 0800 652 6352 or email info@muscular-dystrophy.org

A gift in your Will Did you know that the gifts supporters leave to us in their Wills (however small or large) are vital to fund the work of leading scientists searching for treatments and cures for muscular dystrophy and related conditions? A gift in your Will to the Muscular Dystrophy Campaign sends a strong and personal message of hope for the future.

If the time is right for you and you would like to find out more, please fill out the form below and we will send you an information pack.

Name________________________________________________________________ Address______________________________________________________________ ____________________________________________________________________ _____________________________________ Postcode ________________________ Phone ________________________________ Email __________________________ You can also call Sarah Greenwood for information on 020 7803 4834 or email her at s.greenwood@muscular-dystrophy.org DMN/1203

Contact us at: 61 Southwark Street, London SE1 0HL

020 7803 4834

donations@muscular-dystrophy.org Registered Charity No: 205395 Registered Scottish Charity No: SC039445