MAD CHALLLENGE TEAM NEWSPage 4

QUICK INTERVIEWPage 6

RESEARCH UPDATEPage 2

PoRTRAIT of A SCIENTIST: DR. MARTINE BARKATS

Issue 5 October 2014

NEWSMOMENTUM

Dedicated to finding a cure for Spinal Muscular Atrophy

The SMN gene therapy in US trials today was in fact pioneered by French research scientist Dr. Martine Barkats of the Institut de Myologie in Paris. It uses a harmless virus to carry new genetic material across the Blood Brain Barrier, replacing the defective SMN1 gene. The SMA Trust has funded, either directly or via SMA Europe, Dr. Barkats, or members of her team such as Dr. Sandra Duqué (now working on gene therapy at Ohio State University in the US) and Dr. Yannick Tanguy since 2008. Today and with the help of her growing team, she continues to further refine and improve her product.

At the Families of SMA research conference in Washington, Dr. Barkats presented the work of team member, Aurore Besse, which highlighted the crucial role SMN plays in peripheral organs such as the heart and the liver as well as in motor neurones. Through this new research, funded by The SMA Trust, it has been shown that the lifespan of SMA mice could be significantly increased when SMN is delivered using two different injections: one in the central nervous system and one intravenously (throughout the body, including peripheral organs).

We were all amazed and inspired by achievements like: Octavia’s 120 mile pony trap challenge, Millie’s ‘80 miles without stiles’ or Freddie’s 100 mile UK ‘Coast to Coast’. Since then, many people have done their own MAD challenges and Casimir Knight decided to carry on the tradition this summer by organising a modular Triathlon.

People could:• Cycle a long way in May (100 miles

from Great Ormond St Hospital to The SMA Trust’s offices in Warwickshire).

• Walk a long way in June (Cotswold Way 50, 20 or 10 mile walk).

• Swim a long way.

19 cyclists braved strong headwinds from London, through the Chilterns to the

Cotswolds on 9th May, where they were given a hero’s welcome by The SMA Trust Team. Not satisfied with 100 miles, some of them then went on to do the FT London Sportive 2 days later. Others were up at the crack of dawn to pit their skills in a team clay pigeon shoot (also organised by The SMA Trust).The bike ride and clay shoot together raised over £44k and, although money is still coming in from the Cotswold Way it looks like our summer event series will reach £65k Congratulations and thanks to Cas and fellow Trustees, as well as everyone who hosted, helped organise or took part in these events, not to mention the people who sponsored them in their MADness!

THE fAMoUS fIVE RIDE AGAIN: ALL-SUMMER TRIATHLoN

This phenomenal total goes towards our Muscle Power Campaign, launched earlier this year, which has already raised over £300,000 of the £1 million 2-year target but has a long way still to go.

The fund is enabling us to:• React quickly to fund projects which

speed up and improve the clinical trials process for new drugs.

• Continue our support of UK Centres of Excellence.

• Fund research into new areas that aren’t yet in the clinical pipeline.

• Help the overall understanding of Spinal Muscular Atrophy.

Thank you for all you have done to help.

WITH THE ExCITING NEWS THAT SMN GENE THERAPy STARTED PHASE 1 CLINICAL TRIALS IN THE US IN THE SPRING, WE THoUGHT WE WoULD HIGHLIGHT THE RoLE PLAyED By THE SMA TRUST.

As sOME Of yOU MAy rEMEMbEr, iN 2011 fivE kids wiTh SMAlAUNchEd ThE hUgEly sUccEssfUl MAD ChAllenge sEriEs.

The SMA Trust funds 70% of Uk charity-funded SMA research

www.smatrust.org

All milestones so far have been reached and the project is progressing well. SMA patient populations have been identified at Great Ormond Street Hospital and Newcastle and new exploratory functional scales are being tested. In addition, the team has piloted a standardised physiotherapy assessment database for SMA patients allowing the recruitment of patients to begin in London.

These developments have allowed pharmaceutical company Biogen/ISIS, to choose the UCL Institute of Child Health/Great Ormond Street and the MRC Centre for Neuromuscular Diseases at Newcastle for assessing the efficacy and safety of potential SMA therapy SMN-Rx in infants (see SMA Drug Pipeline chart below).

THE EDINBURGH TEAMProfessor Tom Gillingwater – Developing Novel Small Molecule-Based Therapies for SMA

The Edinburgh team is looking at the molecular pathways known to be regulated by SMN, disruption of which contributes significantly to SMA. The team has also been looking at the potential therapeutic effects of a drug known to target these pathways.

RESEARCH AcTiviTiEs

www.smatrust.org2

Current project updateSMA REACH UKProfessor Francesco Muntoni – Improving Standards of Care and Translational Research in SMA

This project (funded by The SMA Trust through Great Ormond Street Hospital) aims to establish national agreement on medical and physiotherapy assessments and standards of care for patients with SMA in preparation for UK clinical trials.

EVENTSRare Disease DayWe attended an event at The Royal Holloway, London, as part of the 8th Rare Disease Day, an international initiative to raise awareness of rare diseases and their impact on patients’ lives.

FSMA Research Conference & nIh/nInDS Workshop, Washington (June 2014)We attended this 3-day conference which had a heavy timetable of presentations and poster sessions, showcasing new research, as well as satellite meetings with industry and other SMA stakeholders.

UK INITIATIVES

NEW AWARDDr. Heidi Fuller (Fellowship) – The Molecular Consequences of Reduced SMN in iPS-Derived Human Motor Neurones

Dr. Fuller is looking at the differences seen between the human skin cells currently used in pre-clinical therapeutic studies and the motor neurones made from SMA patient stem cells.

The drug, quercetin, has significant therapeutic potential when given to zebrafish, fruit-fly and mouse models of SMA, reducing the breakdown of motor neurones and wasting away of skeletal muscle.

This work was published in the Journal of Clinical Investigation, which resulted in a press release in March 2014, leading to national coverage.

Two exciting new UK projects jointly funded with the Muscular Dystrophy Campaign, totalling £200,000+ over the next 3 years. See website for details.

dr heidi fuller

Danielle Ramsey, ReseaRCh PhysiotheRaPist sma ReaCh UK

RESEARCH AcTiviTiEs

www.smatrust.org

THE ToRCH CENTRE, RJAH oRTHoPAEDIC HoSPITAL, oSWESTRyProfessor Glen Morris, Dr Heidi Fuller and colleagues: The visit was centred around Dr. Heidi Fuller as she establishes herself as an independent SMA research scientist. Her interests are two-fold:

1) Using biochemical and proteomic techniques (proteomics is the study of protein structure and function), she has been looking at SMN to determine how the lack of it affects the whole spectrum of protein activity in cells.

OUr TOUr Of ThE MAiN Uk SMA rEsEArch lAbOrATOriEsis NOw wEll UNdEr wAy, ThE AiM bEiNg TO fiNd OUT MOrEAbOUT cUrrENT prOjEcTs ANd fUTUrE plANs sO wE ArEiN A bETTEr pOsiTiON TO priOriTisE OUr AcTiviTiEs.

2) The effect that SMA drugs have on SMA cells. One potential therapeutic agent under investigation is the protective role of plant polyphenols found in red grapes, green tea and Turmeric.

EDINBURGH UNIVERSITyProfessor Tom Gillingwater and his team have developed a two-pronged strategy on SMA: 1) To understand the disease, using imaging microscopy and molecular approaches to ask what is driving the disease, which tissues are affected and when the disease is active.

2) To develop therapies for SMA. They have had a major breakthrough by identifying the role of the ubiquitin pathway in SMA and the effects of quercetin.Tom has a dynamic team of 11 researchers, at different stages in their careers and whose enthusiasm is contagious. We were fortunate to be able to meet and speak to 5 of them.

Tour of SMA Research laboratories

Annual meetings for both the SMA Europe Board and Scientific Advisory Board (SAB) took place in March, in Munich. Research projects were selected and new initiatives and strategies were agreed.

The Board was also delighted to welcome Fundacja SMA, the Polish organisation for SMA.

SMA EURoPE NEW CLINICAL TRIALS PREPARATIoN fUNDIn addition to the projects, the SMA Europe Board, set up a separate €200,000 Clinical Trials Preparation Fund, to speed up clinical trials initiatives such as specialised workshops for addressing bottlenecks or key issues.

PARIS MEETINGS The French SMA organisation, AFM, kindly invited us to visit them in Paris on 15th & 16th July for a series of presentations and meetings, both in the Institut de Myologie in Paris and at the Génopole ‘bioparc’ in Evry. Discussions with the pharmaceutical industry also took place as part of a European roll-out of clinical trials.The SMA Europe Board took the opportunity to have a mid-year strategy meeting to update priorities in this fast-changing research environment.

lAUNch Of ThE 7Th CAll fOr SMA rEsEArch prOpOsAls

Our 7th Call for research proposals was launched on 1st August 2014 with an application deadline of 16th October. We have advertised this widely so as to attract a good number of outstanding project proposals. For more information, please see the SMA Europe website www.sma-europe.eu.

some of oUR saB memBeRs

sma ReseaRCheR, ewoUt GRoen

THE 6TH CALL foR SMA RESEARCH PRoPoSALSThree SMA projects were selected for funding from 17 applications reviewed. These awards, totalling €390,000 were made to:Dr. George Mentis of Columbia University, New York, will be looking to see if the death of the motor neurones in SMA is due to changes in the cell itself or if it is due to a dysfunction in the network in which the cells belong.Professor Eduardo Tizzano of the Hospital de la Santa Creu I Sant Pau, Barcelona, will seek to understand why the ‘back-up’ gene, SMN2, does not work properly in SMA patients and to find means to induce this gene to produce active protein. They will also be looking for compounds to modulate the intermolecular interactions that promote the production of non-functional SMN protein in SMA patients. They hope that the results obtained will represent an important step forward towards the design of safe and effective drugs for SMA treatment.Dr. Amparo Garcia-Lopez of the University of Geneva, Switzerland, was awarded a Fellowship grant to investigate a molecular structure called TSL2 which regulates the ‘back-up’ gene SMN2. It is known that when this structure is destabilised, it leads to SMN2 behaving like SMN1, producing full length functional SMN protein. Dr. Amparo will therefore be looking at using TSL2 as a drug target.

The child of two carriers has a one in four chance of developing SMA

3

www.smatrust.org4

Having finished 2013 with The Santa Run, a charity football match (Team Travis) and choir concerts (Team Rowan), the new year got off to a great start.

fEBRUARyFebruary saw Team Caoilte arrange raffles, collections and bag packs. We welcomed Team Archie who has done a number of fundraisers, cake sales, Karaoke nights, runs, etc. Team Rowan held bag packs and bucket collections and Primetime staff ran in the Huddersfield 10K.

MARCHThe SMA Trust held a metal detecting day on Compton Scorpion Estate, a new event for us, which raised £3K. Team Arthur held a cake sale, Rapid FX chose us as their charity to support and did a 24 hour Spinathon, raising £3K. Joe Kambanis ran the Naseby Half Marathon for Team Archie. Teenagers from Chipping Campden School helped us with a photo shoot to launch our Onesie Challenge.

APRIL Team Arthur had a great presence at the Virgin London Marathon and raised an amazing £7.5K. Holly Terry from Team Jaiden decided to take on lots of MAD challenges – including Born Survivor, Sunderland Half Marathon, Sunderland Colour Run, Total Warrior, 12 Mile Tough Mudder and the Great North Run! Phew,

May was the start of the All Summer Triathlon, Cas Knight from Team Benny and friends cycled from GOSH in London to The SMA Trust HQ in Warwickshire. It was a beautiful day and several people stayed overnight to take part in our Clay Shoot held on the Compton Scorpion Estate the next day.

JUNETeam Archie – David Rowe held a charity football match. This was the month for our 3rd Cotswold Way MAD Challenge. Julie Smyth started her own fabulous take on fundraising for Team Marni: to celebrate her 50th Birthday, she decided to fundraise by having a glass of champagne individually with 50 friends. Michelle Shaddick and friends from her running club took part in the Torbay Half Marathon for Team Arthur. Frances Brook from Team Abigail ran the Lindley 10K. Team Caoilte – Kelly Nugent organised a fantastic Zumbathon.

JULyGavin Meehan jumped out of an airplane and did a skydive for Team Lucas.

Holly we feel exhausted thinking about that! Rapid FX helped us launch The Cotswold Way MAD Challenge. Team Rowan organised a children’s Onesie Walk. What a great event – such fun and even Tigger turned up! Team Lily-Bea held a fundraiser in Essex, inviting family and friends along. This was another of our ‘meet the Teams roadshows’. We hope to get to do many more over the coming months – do let us know if you’d like to host an event. Christine Blair from Cathy’s Angels organised her annual ball in aid of The SMA Trust, and on the other side of the world, John Brunkard did the Singapore Triathlon.

MAyTeam Caoilte and the Odyssey Bowl staff ran the Belfast Half Marathon, as well as arranging bag packing and football matches. Team Rowan’s little Brook Bissett (aged 2) collected a mile of pennies, raising over £1,000! Gavin White from Team Octavia did 2 back to back cycle challenges, Land’s End to John O’Groats (950 miles) then Venice to St Moritz (400 miles), both phenomenal MAD challenges raising £16K – WOW! Aimee Jewell and Ben Rennick ran the Plymouth Half Marathon for Team Arthur. Rachelle le Bourdon ran for Team Marni. Katharine Jackson (Team Millie) celebrated her 50th Birthday with all gifts coming to us as donations. Team Rowan’s yearly Onesie Walk, saw rain but it did not deter our amazing fundraisers in Yorkshire.

fUNdrAisiNg UPDATE

MAD CHALLENGE TEAM NEWSWE HAVE BEEN VERy BUSy THIS yEAR WITH NEW CHALLENGE EVENTS, NEW fUNDRAISING TEAMS AND GENERALLy DoING WoNDERfULLy MAD THINGS.

WE WELCoMED SEVERAL NEW TEAMS THIS yEAR:Team Abigail (Yorkshire) – in memory of little Abigail Hawtin.Team Amirah (Derbyshire) – Amirah Azam who has SMA Type 2 and her sister Ameenah head up this new team.Team Archie (Northants) – Kay Kambanis (mum to Archie) together with her friends and family started fundraising this year.Team Arthur (Devon) – dynamic team who has done amazing things to support and raise awareness for us this year. Sadly little Arthur died in May but friends and family are continuing their support for The SMA Trust.Team Caoilte (N Ireland) – great fundraisers, they have done many events.Team George (Herts) – Liz Lockley, mum to George kicked off their fundraising with the British 10K this year.Team Lucas (Yorkshire) – organises The Trick or Treat Ball yearly.Team Oscar (Yorkshire) Team Patrick (Lancashire)Team SMAshers – Anna Brooks (Herts)

JUlie & maRni smyth, team maRni

The sanTa run 2013

The Team rOWan Onesie WalK

Joe Kambanis

fUNdrAisiNg UPDATE

CoTSWoLD WAy MAD CHALLENGEThe weekend of June 28th and 29th saw our 3rd Cotswold Way MAD Challenge. 21 walkers chose our 50 mile through the night challenge, 3 joined us at Winchcombe for the 20 mile hike and another 27 at Stanton for the final 10 miles. Money is still coming in, and we still hope to beat last year’s total of £30,000!

BRITISH 10K LoNDoN RUN 11 runners enjoyed this year’s British 10K London run, runners from Team Millie, Team George, Team SMAShers along with the Lanes and Stephen Wickenden for the third time.

THE SMA TRUST GETS SPoRTy!We’re very excited to have been chosen as charity sponsors for 2 teams:•The Yorkshire Jets is one of the top

national netball teams, with SKY coverage of many of their matches. This opens up lots of new opportunities, including the possibility of a celebrity match later this year.

• Team Arthur, Patrick Hawke (Devon) is changing his football team to: United for SMA.

fooTSIE CAMPAIGNOn 1st August we launched our digital media campaign. We asked you to ‘put one foot forward for SMA research’ and take a picture of your foot, post it online and make a donation. This has been a huge success and we have been delighted to see so many pictures and donations coming in. To date, we have raised over £500. Thanks for your #footsie4SMA support.

Get involved...TAKE PART IN A oNESIE CHALLENGE!We would love to see schools, offices and smaller groups of friends doing their own Onesie Challenge this year, a fun walk or run, even a cycle but wearing a onesie!

MAD CHALLENGESThere are many MAD challenges around, marathons, half marathons, triathlons, 24 hour treks and amazing foreign challenges like the Great Wall of China Trek. Why not sign up for either 5k or 10k in this year’s Santa Run in London on 7th December (see website for details).

SCHooLSThanks to all the schools who have done fundraising challenges this year; dress-down days like the children from St. James’ & Ebrington C E Primary in Chipping Campden, cake sales, collections and Onesie walks. Thanks also to Long Ridings Primary School in Essex for making the most of the new Loom band craze and organising a sale for us.

Perhaps we could see some schools taking on a Onesie Challenge soon, the children would love this fun idea.

CoRPoRATERapid FX, a local Gym and fitness centre, has chosen us as their charity to support. They kicked off in May with a 24 hour spinathon which saw several members donning lycra and raising £3,000. Club owner Tim Spittle started the ball rolling with a 1 hour spinning class. Food and drink was provided by local stores including Budgens of Moreton in Marsh.

www.smatrust.org

DUDLEy BALLPaula and Toby Flatman and Hannah McKeown surpassed themselves again this year at their second Ball to raise awareness of SMA and funds for The SMA Trust. The number of SMA families attending had doubled since last year and it’s obvious why. There is a tangible bond between all the families and a real opportunity for the children with SMA to get to know each other. Hollie (Flatman) and Tiegan (McKeown), already firm friends, impressed us all on the dance floor, spinning furiously in their wheelchairs, hardly surprising with such a fantastic band. A fabulous evening all round and over £7K was raised for SMA research. A huge thank you to all involved.

SUPER HERoES Hannah Razzell (Team Lily-Bea), Emma Alderson & Marion Lavocat donned Wonderwoman outfits for the annual Superhero event.

1 in every 6,000 births is affected by SMA

The COTsWOld WaY mad ChallenGe

the BRitish 10K lonDon RUn

Have you signed up for Give as You Live yet? It’s easy and for everything you buy online The SMA Trust receive a % at no expense to you, visit www.giveasyoulive.com.

Give as you live...

The COTsWOld WaY mad ChallenGe launCh

www.smatrust.org

ThANks TO JonAThAn ANd PATRICIA Dee, wE hAd ThE wONdErfUl OppOrTUNiTy Of hOsTiNg A driNks rEcEpTiON AT 11 dOwNiNg sTrEET iN jUly.

We were blessed with a beautiful evening and, as well as seeing the interior of No.11, guests enjoyed the opportunity of mingling and taking photographs in Downing Street itself.There were speeches by Joanna Mitchell, SMA Trust Executive Director, who talked about Spinal Muscular Atrophy and the work of The SMA Trust, followed by Jonathan Dee, who gave an emotional and powerful account of his son David’s life with SMA, his family and friends’ tremendous fundraising efforts (under the banner of Team David) and his hopes for the future. Lastly, Baroness Campbell, campaigner and advisor for disability reforms in the House of Lords, provoked both thought and laughter with her speech, which stressed the importance of supporting people living with SMA (whose bodies are weak but whose minds are unaffected) and enabling them to achieve their full potential. We were thrilled to be joined by George Osborne, Chancellor of the Exchequer and his wife Francis, as well as celebrities including Emily Maitlis and Bill Turnbull, together with Orlando Bloom.Many thanks to all our guests and especially Jonathan and Patricia Dee...and, of course, to The Chancellor and all the 11 Downing Street staff for making us feel so welcome.

HANNAH MCKEoWN, MUM To TIEGAN WHo HAS TyPE 2 SMAQ. How old was Tiegan when you found out she had SMA?A. Tiegan was 13 months when she was diagnosed. We saw the paediatrician on her 1st birthday and waited 4 weeks for the blood test results.

Q. How did you feel? What was your first reaction?A. I was initially informed it was Type 1 so my reaction was heartbreak, shock and confusion, as she didn’t look unwell to me, just weaker than other babies her age. However, she could sit unaided and it was later confirmed she had Type 2.

Q. Can you describe what a typical day is like for you and Tiegan?A. Wake up, breakfast in bed, toilet and shower using Flamingo chair, clothes, hair and cough assist. Then Tiegan uses her lift to get downstairs. Usually it’s school but on weekends and holidays we go to the park, swimming or the gym. Tiegan is a member of Kids Club where they do cheer-leading, mini-dance and creative sessions.

Q. How do you manage to be so positive?A. It’s important for me to be strong as, if I’m down, Tiegan picks up on it. There are so many things to be grateful for so I just focus on those instead.

Q. What are your biggest challenges?A. Biggest challenge is definitely the lack of sleep...whether worrying about things or getting up to make Tiegan comfy. She has a full-on list now including head down, bum, back, sheet up, roll over, arms out, drink, toilet...and I totally get grumpy, but hey that’s what Mums are for!

Q. What are Tiegan’s biggest achievements? What does she like doing the best?A. She was so strong pre and post-surgery, powering through recovery and not letting

anything hold her back. Tiegan is very creative, she loves drawing, painting and ‘inventing’ things and most recently she has taken up Power Chair football. Everything she does is an achievement to me.

Q. When and how did you hear about The SMA Trust?A. A friend of the family told me about The SMA Trust and she did a sponsored swim of Lake Windermere (very proud!). Raising funds for SMA research/support is a part of our lives now.

Q. You are great friends with the Flatman family (Team Hollie). How did you get involved with the Dudley Ball?A. A work friend knew Paula and Toby and recognised that Hollie had been diagnosed with SMA like Tiegan so put us in touch. We met up to talk all things SMA, the girls got on great and the rest is history. I was very impressed with how Paula and Toby organised the first ball within months of Hollie’s diagnosis and was happy to offer my support and help. Tiegan loves any event where she has lots of attention...and rightly so, she’s awesome!

Q. What would Tiegan like to be when she grows up?A. A nurse...but ‘one who teaches other people how to do stuff’. She is clearly aiming straight for management!

Q. What would be the greatest MAD challenge (fundraiser) that you could face?A. Other than next year’s biggest and best Ball yet...would have to be something like The Muddy Run because it sounds like great fun!

QUick INTERVIEW

6

“Baroness CampBell provoked Both thought and laughter with her speeCh, whiCh stressed the importanCe of supporting people living with sma.”

A starry evening at 11 Downing Street

SMA is the leading genetic killer of infants and toddlers

the fast-changing world of sma and the sma trust...

www.smatrust.org

AcTiviTiEs UPDATE

Joanna Mitchell, Executive Director, The SMA Trust

7

To SAy IT’S BEEN A BUSy yEAR WoULD BE AN UNDERSTATEMENT.There is a tangibly heightened air of momentum and positivity within the international SMA community as recent clinical trials show potentially encouraging results.

As a result of the trials, together with other developments, Europe and the US have moved into a new phase of even greater collaboration, not just within the scientific community, but amongst research charities, patient advocacy organisations, pharmaceutical/biotech industries and medicines regulators on both sides of the Atlantic.

This collaboration extends, not just to the current range of potential therapies and how and when to administer them, but also to the drive to extend research programmes to look at factors such as improving understanding of how SMN protein works, its effect on muscles and how SMA also affects other organs and pathways in the body.

This will lead to future treatments that not only improve the lifespan and quality of life of those living with SMA, but also eventually to a cure for SMA.

The SMA Trust is responding to recent developments by becoming even more of a hub for SMA research in the UK and Europe, as well as driving forward new partnerships with US organisations and other relevant SMA stakeholders, wherever they may be.

Our SMA REACH project through Great Ormond Street Hospital is well under way, with plans to extend it over coming months. It has already exceeded expectations in terms of the number of patients recruited. What’s more, the measurement scales that have been developed are now being shared within a series of international workshops, with a view to extending them and ensuring much more consistent and standardised patient data, which will not only help clinical trials, but potentially future care standards.

At the Annual SMA Europe Meeting in March we not only agreed to fund a series of new projects within our Annual Call, but also established a new Clinical Trials Preparation Fund, which would be open to applications from all over the world.

There has also been plenty of change close to home. We recently launched our new website and have also purchased a new supporter database.

In July we welcomed Ginny Cullen as Trusts, Corporate & Major Donor Fundraiser and we also welcome new Trustee, Tom Allison, this Autumn. Both bring a wealth of skills, experience and contacts which will hugely benefit the Trust.

We have also moved into our new offices in Atherstone on Stour, just outside Stratford-upon-Avon. We have

been very grateful to Andrew Knight and the Compton Scorpion Estate Team for providing a home for so long, but had definitely outgrown the space and are now enjoying settling into our new surroundings.

As you can see, the year so far has brought some wonderful and highly successful events, including a very special evening at No.11 Downing Street last month, where we were thrilled to be joined not only by The Chancellor and Mrs Osborne, but also Baroness Campbell and celebrities including Orlando Bloom, Emily Maitlis and Bill Turnbull.

We have been delighted with the support for our Muscle Power Campaign, which has already raised over £300,000 of the £1 million 2-year target and which is already helping us to fund many of our exciting new research initiatives.

Finally, we are, as always, tremendously grateful to all the wonderful people who have given their time, money and, most particularly, energy over the past few months. Their energy reflects the energy that currently exists in SMA research. The SMA Trust is committed to playing its part in ensuring it continues and is channelled in the right directions – leading us along the path that leads to a cure and treatments for Spinal Muscular Atrophy.

Thank you for everything you do to make it possible for us to do that.£300k

of THE £1 MILLIoN TARGET HAS So fAR BEEN RAISED By yoUR SUPPoRT of oUR MUSCLE PoWER CAMPAIGN

1 in every 40 people is a carrier of the defective gene that causes SMA

GET IN ToUCHExecutive Director Joanna Mitchell joanna@smatrust.orgResearch Co-ordinator Vanessa Christie-Brown vanessa@smatrust.orgCommunity & Events Fundraiser Mandy Lane mandy@smatrust.orgTrusts, Corporate & Major Donors Fundraiser Ginny Cullen ginny@smatrust.org

The SMA Trust 1c Atherstone Barns, Atherstone on Stour, Stratford-upon-Avon, Warwickshire CV37 8NE Telephone: 01789 801155

twitter.com/smatrust facebook.com/smatrust www.smatrust.org

Registered Charity Number: 1097765

Data Protection: If you no longer wish to receive this newsletter or any information about The SMA Trust, please email mandy@smatrust.org or call 01789 801155.

fUrThEr INfoRMATIoN

We would like to welcome Ginny Cullen who has recently joined the team. Ginny brings a host of experience with her and she will be responsible for trust applications, corporate liaison and major gifts.

how can you help?➜ You can make a regular gift or donation to The SMA Trust, see form below.

➜ Donate directly to The Trust www.smatrust.org/donate/donate-2➜ Do your own MAD Challenge. It can be anything; running, walking, tea parties,

cake sales, car washing, the MADder the better! Come up with your own ideas. For more inspiration or help contact us on 01789 801155.

➜ Volunteer – we are always looking for help either here in the office, remotely or at our events throughout the year. We need local ambassadors too. If you think you can help please contact mandy@smatrust.org or call 01789 801155.

We are just putting together an exciting Calendar of Events for next year, keep an eye on our Facebook page facebook.com/smatrust and website www.smatrust.org.

WELCoME To GINNy CULLEN

Leaving a GIfT IN yoUR WILL TO THE SMA TRUST

Leaving a gift in your Will will help to ensure that we can continue to fund research into finding a cure and treatments for SMA, which is still the leading genetic killer of children under 2.By leaving a gift, no matter the size, you will continue to make a positive impact and motivate change beyond your lifetime. If this is something you choose to do, it is important that our name, address and charity number are featured in your Will:The SMA Trust, 1c Atherstone Barns, Atherstone on Stour, Stratford-upon-Avon, Warwickshire CV37 8NERegistered Charity Number: 1097765Please see our website for more information or contact the Fundraising Team with any queries.

I WoULD LIKE To MAKE ArEgUlAr gifT Or dONATiON

please return this form to: The SMA Trust 1c Atherstone barns, Atherstone on stour, stratford-upon-Avon, warwickshire cv37 8NE

1 3

4

yOUr dETAils

Title: first Name:

last Name:

Address:

postcode:

Telephone Number:

Mobile:

Email:

2 MAkE A rEgUlAr gifTi would like to make a regular gift of

£

starting on

and the same sum on the same day monthly

Please set up a standing order with your bank to:

lloyds TSB, Stratford upon Avon (Sort Code 30-98-26)

The SMA Trust (Account no. 02628431)

signature:

date:

MAkE A dONATiONi would like to make a one off donation to The SMA Trust of

£

i have enclosed a cheque made payable to The SMA Trust

gifT Aidgift Aid means that for every pound you give, we receive an extra 25p from the inland revenue, helping your gift go even further.

Yes I want all donations I have made since 6th April 2008, and all donations I make in the future, to be eligible for gift Aid. please tick the box above.

thank you for your support