Campaign Newsletter April 2015

Campaign

The newsletter for our supporters

Inside:

Archie at 10 Downing Street Christmas appeal update Lord Attenborough memorial

BBC One Lifeline Appeal Thank you to all of you who supported the BBC Lifeline Appeal last month. We’ve raised an amazing £32,000 towards research and to support families affected by muscle-wasting conditions. You can still watch the programme on www.musculardystrophyuk.org/lifeline

Jonathan Gilmour, 28, living with Duchenne muscular dystrophy, appeared in the BBC One Lifeline Appeal

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Welcome Welcome to this edition of Campaign, with updates and news of exciting developments and events from Muscular Dystrophy UK. You will have noticed we have changed our name to Muscular Dystrophy UK to reflect the wider reach of the charity beyond campaigning.

The name change will not alter what we do in any way.

We are as committed as ever to finding treatments and cures as quickly as possible, and to offering care and support to those affected to enable them to lead independent lives.

I hope you enjoy reading your Campaign newsletter. It is thanks to your support we can have an impact on so many lives. Thank you.

Sue Barker, MBE

President, Muscular Dystrophy UK

Our vision A world with effective treatments and cures for all muscle-wasting conditions and no limits in life for individuals and families affected.

Campaign newsletter for supporters of Muscular Dystrophy UK, written and produced entirely in-house.

Muscular Dystrophy UK, 61A Great Suffolk Street, London SE1 0BU 020 7803 4800 info@musculardystrophyuk.org www.musculardystrophyuk.org Registered Charity No. 205395 Registered Scottish Charity No. SC039445 DMN/1504

Join us online Make sure you keep up-to-date by joining our social media networks. www.facebook.com/musculardystrophyuk Join the 20,000+ community on our Facebook page. @MDUK_News Keep on top of our breaking news by following us on Twitter. www.pinterest.com/MD_UK Follow our boards and pin our pictures on Pinterest. www.youtube.com/musculardystrophyuk Watch our videos on YouTube. instagram.com/musculardystrophyuk Share our pictures on Instagram.

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Update on Harry

Harry Barnley is living with Duchenne muscular dystrophy

Thank you to all our wonderful supporters for responding so generously to our Christmas appeal. Together you’ve donated over £30,000 towards research to find cures and treatments for muscle-wasting conditions.

You may remember Harry from our Christmas appeal? Harry was diagnosed with Duchenne muscular dystrophy at the age of five. We recently caught up with his parents, Sue and Tom, and this is what they told us.

“Harry is becoming much more aware of his condition now and this is something we’ve had to tackle. We started by just talking about his legs, normally when his brother Jack was around as well. Just saying things

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like ’we are all different, some of us have to wear glasses, some of us are not very good at running ... we’re all different’. We told Harry his legs were different and not as strong as other children’s. He asked if that was why he was slower. We often ask how his legs are, just getting him thinking about it more and more. We’ve told him he might need a bit more help as he gets older.

“Now Harry knows he will need to be in a wheelchair one day and has even started to point out what sort of chair he wants.”

Harry has also outgrown his old night splints (or ’night wellies’ as Harry and his family call them) and has got a new pair – leopard print this time!

“Harry is becoming much more aware of his condition now and this is something we’ve had to tackle.”

exercise weights along with them and let the children put them on to run around in. Again it will give them a feeling of what it is like for Harry.

Soon the family will go to Harry’s class to explain what it is like for Harry to live with Duchenne muscular dystrophy – so his friends understand his condition and can help him. They’ll ask children to imagine wearing a suit of armour for the whole day. They’ll say it might sound fun at first, but that’s what it’s like for Harry all day, every day.

They will take some wrist and ankle

Sue and Tom are pushing for more research to be done into Duchenne muscular dystrophy and raising money for the charity. They have set up help4harry as a Family Fund, which you can find on our website.

If you would like to find out how you could set up a Family Fund like help4harry, go to: www.musculardystrophyuk. org/familyfunds

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Research event in Scotland

A young researcher studying live cells

Recently, Muscular Dystrophy UK-funded researcher, Professor Thomas Gillingwater, hosted a fantastic day for our supporters.

They visited his laboratory at the University of Edinburgh to look behind the scenes.

Our supporters were taken on a tour of the laboratory and had the chance to observe live cells being grown in culture under the microscope.

John Veene, whose daughter has congenital muscular dystrophy, said, “Looking around a research laboratory was a wonderful experience. Talking to scientists and seeing the work that goes on in a laboratory has really shown me how much effort and learning goes into the research we are funding.”

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Professor Gillingwater, whose research is partly funded by the charity, said, “We are very grateful to Muscular Dystrophy UK for funding our research and we are delighted to be able to show some of the people who have worked so hard to raise this money around the laboratory.”

If you would be interested in finding out more about our research programme or attending a future laboratory visit, please visit our website www.musculardystrophyuk. org or call 020 7803 4800.

Research news Utrophin up-regulation

Exon skipping

Utrophin up-regulation could potentially be a promising treatment for boys with Duchenne and Becker muscular dystrophy.

Researchers have been screening compounds that up-regulate utrophin in the laboratory.

Muscular Dystrophy UK funds a wide range of research projects including the exciting and promising work in the field of exon skipping, which is currently on clinical trial for Duchenne muscular dystrophy. Having funded research into this technology for the past 20 years, we are now also exploring how it might be used for other musclewasting conditions.

Summit Therapeutics, a pharmaceutical company based in Oxford, has been testing one such compound, SMT C1100 in a clinical trial. The trial showed that the compound was safe and well tolerated.

Recently the company announced approval from UK regulators to start a new phase 1b clinical trial to test whether a particular diet can increase the uptake of the compound by the muscle. This information will be crucial for planning future clinical trials which will test whether the potential drug can increase levels of utrophin in people with Duchenne and Becker muscular dystrophy.

Muscular Dystrophy UK has been involved with research into utrophin up-regulation for more than 20 years.

To keep up to date with all the cutting-edge research projects Muscular Dystrophy UK is funding, visit www.musculardystrophyuk. org/research

Clinical trials A new clinical trial on facioscapulohumeral muscular dystrophy (FSH) has shown that vitamin supplements may have a small beneficial effect on the progression of the condition. However the results of the trial are very preliminary and supplements should not be taken without medical advice, as they may be harmful.

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Archie’s big day

Archie and his family meeting the Prime Minister while delivering a petition on speedier access to Translarna

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Archie, who’s on the front cover of this Campaign newsletter, was diagnosed with Duchenne muscular dystrophy in 2008 at the age of three. After receiving support and advice from Muscular Dystrophy UK, Archie’s parents Gary and Louisa have been raising awareness of the condition. They have worked with the charity to fund research into finding effective treatments for Duchenne muscular dystrophy, and to push for faster access to potential treatments for children with muscle-wasting conditions.

“Our focus since Archie was diagnosed has been to remain positive and to do anything and everything we can to help. We are hopeful that treatments can be developed that will benefit Archie and other boys affected by Duchenne muscular dystrophy.

Research is progressing and we are keen to help the researchers capitalise on the current momentum and build on the findings,” said Louisa, Archie’s mum.

Archie and his parents met the Prime Minister at a lobby in Parliament on 14 January. Archie delivered a petition signed by 24,000 people to get access to a new potential drug, Translarna, which could slow the progress of Duchenne muscular

dystrophy. On his visit to 10 Downing Street, Archie said his wish came true as he met the Prime Minister.

“Translarna is the first drug that has given us and the whole of the Duchenne community real hope.

We understand it is not a cure but it will slow the progression down and give Archie and other boys with Duchenne more time to enjoy playing with their brothers, sisters, friends and family, something most of us take for granted. As parents we would do anything for our children,” said Gary, Archie’s dad.

Duchenne muscular dystrophy is a devastating muscle-wasting condition that affects mainly boys. It causes muscles to weaken and waste over time, leading to increasing and severe disability. It is caused by the lack of a vital muscle protein called dystrophin. It is thought that there are 2,500 people in the UK living with the condition.

There are currently no known treatments or cures. Few of those born with the condition currently live to see their 30th birthday.

You can find out more on Archie and our #FastForward campaign for access to potential treatments in the UK by visiting www. musculardystrophyuk.org/ fastforward

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Advocacy Ambassadors

Sarah Thompson, West Midlands Muscular Dystrophy UK Advocacy Ambassador, with her son Bradley, 10, who has Duchenne muscular dystrophy

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Here to help you fight for your rights “It gives me great pleasure to do my job as an Advocacy Ambassador for the West Midlands and I am always willing to help any families with muscular dystrophy.”

Mum, Sarah Thompson, became an Advocacy Ambassador to help others navigate the complex health and social care system.

She draws on her own experience of getting support for her son Bradley, 10, who has Duchenne muscular dystrophy.

Sarah has been supporting Nikola’s family through their application for an extension to their home. Nikola’s son, Cameron, has congenital myopathy and the family needed to adapt their home to suit his increasing needs. With Sarah’s support and experience, Nikola and her family were able to successfully get their application for adaptations to Cameron’s new bedroom and the family’s living room approved.

through her own experiences as a parent of a child with Duchenne muscular dystrophy. Thank you.”

Advocacy Ambassador Sarah said: “It was my pleasure helping Nikki and Cameron. I wanted to help work out any problems and to make sure this family did not go through the issues l had when adapting our home. It was important to me to make sure their adaptation plan was appropriate to them as a family.”

Need advice about getting the services, benefits or equipment you and your family are entitled to? Your Advocacy Ambassadors are here to help. They’ve been through it all themselves, so they know just what you might need.

To see how we can help you or to find out how to become an Advocacy Ambassador, contact: ambassadors@ musculardystrophyuk.org or call 020 7803 4800

Talking about Sarah’s support, Nikola said: “Sarah has been very informative and supportive helping me with my dealings with Independent Living. She is very passionate about the cause and offering the right help and support to other families

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Lord Richard Attenborough

After meeting boys with Duchenne muscular dystrophy at a local fête in 1962, Lord Attenborough pledged his commitment to fight muscle-wasting conditions. His passion, dedication and hugely successful fundraising were instrumental in getting us to where we are today.

A service of thanksgiving for the life and work of Lord Attenborough CBE was held at Westminster Abbey on Tuesday 17 March 2015.

Richard Attenborough served as President of the charity from 1972 until 2002.

He offered his support to us after visiting a local fête in 1962 and meeting some boys who had Duchenne muscular dystrophy.

Without his passion, dedication and successful drive to generate funds for research, we would not be as close as we are today to the first potential treatments being available for

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Duchenne muscular dystrophy and other muscle-wasting conditions.

“I’ve received far more than I have given as far as the charity is concerned. The privilege of knowing the parents and, of coming to understand the depths of what their utterly unselfish love can amount to is a gift that perhaps I will never witness anywhere else.

And I shall be ever grateful to Muscular Dystrophy UK for that.”

Lord Attenborough

In recognition of Lord Attenborough’s dedication to the charity, the Attenborough family most generously chose Muscular Dystrophy UK as the recipient charity for donations made at the service of thanksgiving. We are most grateful for this honour, and for the more than £26,000 raised.

Gifts made in Lord Attenborough’s memory will be used to support the Richard Attenborough Fellowship Fund. This fund was established in 2012 in honour of Lord Attenborough’s lifelong commitment to the charity’s groundbreaking research. The fund continues his legacy.

Lord Attenborough CBE 1923-2014

Give in memory of someone special A donation to Muscular Dystrophy UK is a very special way to remember and honour the life of a relative or friend. Should you wish to give in memory of someone you love, you can do so knowing your donation will be used to give hope and support today to families living with muscle-wasting conditions.

If you would like more information on giving a gift in memory, please contact Andrew on a.moran@musculardystrophyuk.org or 020 7803 4823

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Get active

Oxford Town and Gown 10k

Prudential Ride London – Surrey 100

Sunday 10 May 2015

Sunday 2 August 2015

Do you need some motivation to get to the gym or out running?

Why not sign up to the 34th Muscular Dystrophy UK’s Oxford Town and Gown 10k on Sunday 10 May 2015? You will be joining 4,000 runners of all abilities and together you’ll help us turn Oxford orange.

There is still space to register for our Oxford Town and Gown 10k or for the Junior 3k event.

This fantastic 100-mile cycling event takes you down the closed streets of London and Surrey’s stunning country roads and hills for a unique experience. Be part of the biggest and best cycling event in the country.

To register, please visit our website www.townandgown10k.com

We will support you every step of the way, providing you with training and fundraising advice from our experienced team and a branded ‘stand out from the crowd’ cycling jersey to train and cycle in.

Lucky enough to win a ballot place?

Join our #TeamOrange and you’ll still receive all the above benefits.

For more information, please call 020 7803 4820 or contact events@ musculardystrophyuk.org

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#TeamOrange needs you You don’t have to run or cycle to become part of #TeamOrange.

You can help and encourage our runners, who train hard preparing for their big day.

Whether it’s to spot and cheer on our #TeamOrange runners as they make their way through the streets or to welcome them like heroes on the finishing line – we need you.

For more information, please call 020 7803 4820 or contact events@musculardystrophyuk.org

Great Muscle Raffle Thank you so much to all of you who took part in our Great Muscle Raffle. The raffle raised over £15,000, which will be used to fund research and to support 70,000 children and adults with muscle-wasting conditions to live as independently as possible. Congratulations to Mrs Lawrenson who won the first prize of £3,000. Congratulations to Mr Bullman and Ms Taylor who, respectively, won the second and third prizes.

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Gifts in Wills For many children living with muscle-wasting conditions a simple smile and a comforting hug soon become impossible.

Muscular Dystrophy UK has been funding research to find effective treatments and cures, and helping families live with muscle-wasting conditions for over 50 years. Our work depends almost entirely on charitable donations, including gifts in Wills, voluntary donations and family fundraising.

If you would like to find out more about leaving a gift in your Will to Muscular Dystrophy UK, please contact Benedicte Benoit on: 020 7803 4834 legacy@musculardystrophyuk.org

www.musculardystrophyuk.org/legacy Muscular Dystrophy UK, 61A Great Suffolk Street SE1 0BU Registered Charity No. 205395 and Registered Scottish Charity No. SC039445

#MusclesMatter