CHARITY COMMISSION FOR ENfjLAND AND WALES Cure Myotonlc Dystrophy UK Charfty I¢DMI 1191217 Receipts and payments accounts CC16a For the perlod (W2022 0510412023 Section A Receipts and payments Unr8strict8d fund8 Restncted funds Endowmont funds Totsl lund$ LaBi yoor totho to Iho rbTrarost£ toth• n•rnt£ to tho noaMt£ A1 R•co1 tndIn5 29.914 2.801 823 18,475 29.914 Z.B01 823 18.475 Communty Evenis Refunds SponsorshipslGranL% ross income AR) 52.013 52.013 Set an 158e table). nvastmant 8al88, Total rnc•ipts $2.013 A3Pa ment8 munty Evants Adrnlntran & Running ¢08ts pa31 & JustGiving FéeB Re5earth l>anls Conferences Adverjsing & PrOMoal Merthand*e PiolessK>Dal Fees 28,534 11,88B B71 6.270 319 3.557 2.222 28,$34 11.888 71 6270 31 3.557 2.222 Sub fOt•l 53.661 .661 A4 Asset and Inv85tment urchas•s So• tabl• Sub total Totalpayments 53,661 53,e61 Net of receipts/(payments) A5 Tronsf¢rs betwgon funds A6 Cash funds last yèar and Cash fvnds this year end 1,649 47 34,734 CCLX R1 arxwnts Issi 1012023
Socllon B Slalomont of assels and liabilities at Ilie ond of Ilio perlod C•l•9Ofl•• 81 ¢••h fundA J4n• Mn4 d• Ihill• D•i•ll• ehltrf8 own w• . O•iJ>li C£XXP2•xtyk* ISS
CHARITY COMMISSION FOR ENGLAND ANO WALES Independent examiner's report on the accounts Sgction A Indgpgndent Examinerfs R9PQrt Report to the trustee$ Cure Myotonic Dystrophy UK Charity ICDM) On a¢¢ounts for tho year ended 0510412023 Charity no Ilf any) 1191217 Set out on pages 1&2 I report to the Iruslees on my examination of the accounts of the above charrty for the year ended 0510412023. Responsibilities and basls of report As the charivs trustees, you are responsible for the preparation of the accounts in accordance with the requirements of the Charities Act 2011. I report in respect of my examination of the Trust's accounts carried out under section 145 of the 2011 Act and in carrying out my examination, I have followed all the applicable Directions given by the Charity Commission under section 145(5llb) of the Act. Independent examlner's statement I have completed my examination. I confirm that no material matters have come to my attention in connection with the examinatson which gives me cause to believe that in, any material respect- the accounting records were not kept in accordance with section 130 of the ChaTlties Act. or the accounts did not accord with the accounting records., or the accounts did not comply with the applicable requirements conceming the fom) and content of accounts set out in the Charities (Accounts and ReFX)rtsl Regulations 2008 other than any requirement that the accounts give a 'true and fair, view which is not a matter considered as part of an independent examination. I have no concems and have come across no other matters in connection with the examination to which attention should be drawn in this report in Ofder to enable a proper understanding of the accounts to be reached. Signgd: Dalg: 0211012023 Name: Rebecca Rouse TIAS BSR Accountancy Relevant professional quallflcatlon{sl or body lif any): Chartered Institute of Management Accountants ICIMA) CGMA,ACMA,MIP Address: 15 Crakedale Road Winlerton IER Oct 2018
DN159UT Section B Disc105ure Only complete Il the examiner needs lo highlight material mallers of Goncein (see CC32. Independent examination of charity accounts.. directions and guidance for examiners). Give here brief details of any items that the examiner wishes to dlsclose. IER Oct 2018
Cure Myotonic Dystrophy UK Charity (CDM) (CIO) REPORT OF THE TRUSTEES: 6[th] April 2022 to 5[th] April 2023
The Trustees present their report together with the financial statements of the Charity for the year ended 5[th] April 2023.
Reference and Administrative Details:
Charity Number: 1191217 Registered Address: c/o Riverside View, Station Rd, Whitton, North Lincs, DN15 9LR
Directors and Trustees: The currently registered Trustees are:
Emma-Jayne Ashley, Peter Ashley (Chair), Stephen Uncles, Dr Alison Kay (all sitting as first trustees). Liam Garwood and Tamsyn Tait (as of 4[th] Jan 2023).
There must be at least three (3) charity trustees. If the number falls below this minimum, the remaining trustee or trustees may act only to call a meeting of the charity trustees or appoint a new charity trustee. Apart from the first charity trustees, every trustee must be appointed for a term of three years by a resolution passed at a properly convened meeting of the charity trustees.
Objectives, Activities, Achievements and Performance:
The objectives of the CIO are:
To preserve and protect good health among, and relieve the needs of, people living with Myotonic Dystrophy, their families and carers, in particular but not exclusively by:
(a) providing information, help and support to such people and their families and carers;
(b) making financial donations to support organisations and individuals carrying out research into Myotonic Dystrophy, the useful results of which will be published for public benefit; and
(c) raising awareness of Myotonic Dystrophy within the general public, medical and scientific communities.
Raising funds is always high on our priority list. Financial income for this period came from donations and fundraising, the community, sponsorships, grants, and donations from CureDM Ventures CIC. The Board of Trustees have regular updates/meetings, and an annual AGM (which is the main source of future Charity planning).
Services were maintained in the following areas:
The return to in person events, consultations, and conferences. Discussions between all stakeholders were held in person, and via continued utilisation of virtual meetings.
For the period April 2022-2023:
Research
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Presented CureDM Posters at worldwide scientific conferences, raising the profile of congenital and childhood onset Myotonic Dystrophy (DM) in the UK.
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Submitted an abstract and presented a poster at the International Myotonic Dystrophy Consortium (IDMC), worldwide scientific conference in Japan (virtually).
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Co-authored DM papers and posters on Patient preferences, DM1, DM2 and CDM.
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UK DM Data continued to be gathered via ongoing community questionnaire.
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Founding members of Euro-DyMA, an international collaboration of European DM support groups. Euro-DyMA meet regularly online and in person to facilitate research and to define and meet the un-met needs of the DM Community.
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Patient representative supporting the ILAP application process within MHRA, SMC, AWMSG and NICE.
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Advised on DM relevant study protocol/design as patient representatives on numerous ongoing research projects, including direct meetings with organisations working towards clinical trials for potential new treatments in the UK, and DM natural history studies. Vital to find treatments and therapies for those living with Myotonic Dystrophy.
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Using our Subject Matter expertise in Myotonic Dystrophy to provide input, advice and support to wider Neuromuscular committees and panels.
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Steering committee member for the UK Myotonic Dystrophy Patient registry.
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Patient representatives for upcoming potential research studies and clinical trials, advising on the complexity of DM/CDM.
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Collaborated with clinicians and researchers to produce disease specific guidelines and advice. Notably PEoLC for DM1.
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Attended and presented at carefully selected worldwide scientific, medical, and clinical conferences (online and in person).
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Ongoing European Patient Advocacy Group representative on the European Reference Network (ERN) for neuromuscular diseases.
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Provided grants to the UK DM Patient Registry.
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Provided grants to researchers to support attendance and research presentation at Neurology conferences.
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Reviewed and agreed to co-fund UK Natural history study into Adults with Congenital Myotonic Dystrophy (next year’s accounts)
Awareness:
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Joint founders of the INTERNATIONAL MYOTONIC DYSTROPHY AWARENESS DAY.
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Continued to promote the awareness day and collaboration throughout the year.
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Member of steering committee for the DM Global Alliance.
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Designed ‘could it be Myotonic Dystrophy’ posters for dissemination to all settings where patients may be seen. These are now used worldwide.
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Bi-monthly Global Alliance (online) meetings with worldwide support groups and organisations. Global collaboration to raise awareness and bringing all communities, scientific, research, patients, and pharma, together.
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Regularly updating website with accurate, DM specific, information and advice.
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Social media pages including public Charity pages and dedicated private peer support pages, allowing patients and families with DM to connect in private.
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Moderates worldwide social media pages for DM1, DM2 and CDM.
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Advocates for families with DM in charitable, social, and medical settings.
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Attended several online medical, research and support conferences including World Orphan Drug Congress, MDUK, MDF, Genetic Alliance, Eurordis, Euro-Dyma, IMI, WMS, Treat-NMD, CNMD and other rare disease communities.
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Presented at some of the above conferences and manned CureDM information stands.
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Raised the profile of Myotonic Dystrophy in Rare disease, Neuromuscular, Pharma and Muscular Dystrophy focused collaborations.
Support:
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Re-started face to face meetings in July 2022 with a Families Day event at Alton Towers, supporting 20 families and over 70 participants.
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Facilitated group events at Disney on Ice in Nov/Dec 2023, in 3 venues around the UK, for 16 families living with Myotonic Dystrophy.
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Delivered support, advice, and assistance virtually and in person to the Myotonic Dystrophy community, dealing with the queries or passing them on to an appropriate person or organisation.
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Produced ‘medical need for space’ and ‘please give me space’ cards and badges for the community re-entering public as pandemic measures reduced.
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Supported individuals and families with day to day, benefits, healthcare, and quality of life issues.
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Successfully supported community members in applying for disability benefits and home adaptions.
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Continued to provide ‘Miles’ mascots to children with CDM to promote friendship and support.
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Commissioned a “life-sized” ‘Miles’ mascot costume to help with engagement at face-toface community and fund-raising events.
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Supporting community members by providing information and advice to enable them to advocate for themselves and loved ones in medical situations.
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Supported families dealing with the devastating effects of the condition, including during bereavement of loved ones.
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Supports work in advancing Palliative and End of Life provision for the DM community.
Fundraising:
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Fundraising events included online campaigns and personal fundraisers/donations.
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Grants and sponsorships were received to support conference attendance, admin support and the running costs of the charity, enabling us to use personal donations for community support and facilitating research (ringfenced if specified).
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We continued to receive generous donations from people fundraising on our behalf.
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In memory donations received.
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CureDM trustees organised and ran with the team for Great North Run 2022 raising over £9600.
News and information:
The charity facilitates a private social media group, and a public charity page on Facebook. Also utilising Twitter, LinkedIn, and Instagram accounts. CureDM have a YouTube page to share community videos and charity presentations. These are platforms for sharing articles, peer support, sharing experiences and ideas. Information is shared on research progress and other matters impacting families of disabled children and adults. There is also a website used to keep families up to date with news and events. The 2022/2023 Impact Report was published and is attached to this Trustee report.
Stakeholders:
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Charity stakeholders are those affected by Myotonic Dystrophy, their families and carers, those involved in running the Charity, volunteers and those fundraising or raising awareness, the clinicians, researchers and drug developers that engage with and benefit from our work.
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Support, events, and fundraising opportunities are proposed and discussed by stakeholders. Consultations take place regularly in meetings with families, during business meetings and conferences and online via e-mail and social media.
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When deciding on future peer support get-togethers, the activities, meet up plans, venues, dates, and itineraries are planned around consultation with the potential users themselves. Plans are made to include as many people as possible, whilst making efforts to reduce attendee bias and ensure those who have not been able to attend previously are accommodated.
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Day-to-day decisions are made with consultation between trustees, volunteers, and community members.
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Strategic or significant financial decisions, such as activities or proposals for research funding, are made following consultation with all trustees, who all have significant knowledge and experience of the conditions, support for others, charitable work, and fundraising.
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Research/Grant funding requests are approved following consultation with the trustees to determine the most appropriate use of funds.
Events:
The charity endeavours to facilitate social events where families can enjoy a relaxed atmosphere whilst sharing their experiences. Events are aimed to facilitate peer support, family memories and encourage the sharing of knowledge to improve day to day quality of life.
Review of Public Benefit:
In setting out the objectives and planning activities, the Trustees have carefully considered the Charity Commission’s general guidance on public benefit. Face to face events were reinstated with provision in place to keep community members as safe as possible after the COVID-19 pandemic.
Future Plans:
CureDM continually strives to expand services to meet growing demand, in a planned and financially prudent way, ensuring all projects stand on a sound financial footing and that the Charity moves forward and grows, but does so responsibly.
Income/Expenditure:
The restrictions necessitated by the Covid-19 pandemic and the vulnerability of the community/families caused a delay in planned social/peer support activities (days out, holidays etc.), normally at the core of the Charity’s work. Careful planning allowed these events to begin again, showing as an increase in expenditure for this financial period.
It is the belief of the Charity that funds should be used to fulfil the charitable aims as available, and not to accrue a large unused credit which could otherwise be used to benefit the community we serve.
Investment powers and policy:
The CIO has power to do anything which is calculated to further its Objects or is conducive or incidental to doing so , as laid out in the Constitution.
Reserves policy:
The state of the economy and resulting downturn in donations received means sufficient reserves may need be held to ensure the future running of the charity. There is no policy to date in place dictating reserve amounts. The Trustees carefully monitor the finances to ensure adequate funding is available for the continuation of services and financial obligations.
Structure, Governance and Management Governing Document:
Cure Myotonic Dystrophy UK Charity (CDM) is a registered charity (charity number 1191217), which achieved registered charity status on 9 September 2020.
The CIO is governed by Constitution of Charitable Incorporated Organisation (Foundation Structure). The CIO is registered with the Fundraising Regulator.
TruMes ire appolnted Trusiees at the Amual General Meetlrrf In ac(OfthKo ¥Ath tNe Chrivs CarlIt. No enernal bodles the d8ht to appdfitTrustees. tralnl New Trurtee5 •re 1fitr0d to the operauons olthe ory•nlMtkn Ind •fo thn InlomHtloft on the responbIlItIeS ol beln8 a TNstee •s tll •> coples ol the ConMltUllo Tralnlnq Is Prtyled Trustee's a5 requirEd. The bo•rd ol TNstees mett rewlarly and adffllnlsier the CIO. Th• th•fltyhs ro empl•$. Tht Trustees Ye4xe for •U Iled51on TrUse5. re$F#)ftbl11tles requlrn preparatlon of Ilnindal staitments Ihat i true Ind lllrthof the stste of affalrs of the CIO. •t the end of the firlI1 year, and of Its surplus Of defidt for the fin•Thd•l yoar. In dolry so ihe Tn1¢$ ère rwulfed to: select suiuble aCcoUnnI poNcles •nd then apply them coftslstenily ¢)bser¥e the mrthods and prlndples In the Ch•fftlesSOAP Istatsrnent ol ¢ec¢rn make Judyments and estlmètes that are lsOn1b1¢ •fid pnAert stsfe whethef applicable accounting stsndafds have been follv•ed, SleCt to iny rn•teil•l dewrtures dlsclostd and explalned In the Ilnantlal ststernents prepare the finandal slatementson the Bolng concern baslL The Trustees Jre respon51ble for malntalnlng accountSng records whlch dlsdose wlth rea50ble •eturntyat ary tlme the flnandal poslik>n ofthe ao. The Tfustees are also responslble for Safe8rdI111 tht assets ol the Chrlty, tkln8 reasonable steps forthe thntIOn and deleclkn ol fraud and other Ifrn8ulaTltJe& 8ned by two tntstee5 on behalf of all trustees: Trustee I: Sr4Y% A- Name ind date: 06 . Ko. Trv5tee 2: Name and dale: 04/Jè/
th2023 A Snapshot of What We Got Up To This Year I DM CureDM is a small yet mighty collective of families, carers, loved ones and special individuals. We would like to thank everyone who makes CureDM charity what it is. Cure DM Support V Awareness v Research We've seen our donations almost double in 2022/ 23 and our expendiiure has increased to reflect this. This simply wouldn't have been possible without your support! We would like to sincerely give thanks to those whose efforts, conlTibutions, and donations - big and small- make up the CureDM community. All the Trustees on our panel have the unique perspective of being parents and carergivers to a chiId/ young adult diagnosed WLth Congenital Myotonic Dystrophy or a similar neuromuscular condition... Emma-Jayne Ashley Foundin8 Director & Trustee Mum to Dregan Stephen Uncles Trustee Dad to Matthew & husband to Paula ICDM & DMI) Peter Ashley TnsstÈe & Chair Dad to Dresan {CDMI Dr. Afii Kay TTUStee Mum to BLirtie ILQlrith CMDI ICDMI In 2022 we gave a warm welcome to two new additlons to the CureDM Trustees... "l ttlii rertlly L'x¢itÉd to join tlte CurL'DM "I'm delislited to be joining t CitreDM teoFii. My fvvfr.yellr-old knuxliter's teain. My one-yLwr-olil 11 uirt dtft8n(t.S of CDMI llnd lliyfflvn dth%nosÉd ttJitlT CDM sIrElyer biTIIT -%ub5eqiient dtrtsno%is of DMI I ulong with niy wtfe (DMI). Tlie news reltttÉ lo tljose ttylTO Imve tl QTyiie u5 a17U8e.siiock, it dog5for yNany comdilion wliil41 rnrijisfvr n rhildwith frmilie4. 1 lookfvnvfird fro CONtributiK% Ilr %everefvnJi. Willi fi cnrger175 to thE cIIftTity 5fllntllsfric i Mlltretiiis MttnAger, I'm Ixyiitg 1 ts7n IlY Flie C117tY,$PrOfj}¥ ultriltirttely tttLJQTen¢ssfvT tITÉ discrtse. I Jii H150 keen to support oiher5 tlirough lIr journey." (Learn more about the team by visitins our web site) Tamsyn Tate awftrene.q witliin t1.lence¢lllA meilicftl coniintsnity to lliovi, tottmrds efrctive trel71Nnt.~ Trustee Mum to Daisy Lia uie, ICDM & DMII d to Bayley & DMII Ic PJge1
Ongoing Support This year, like every year, we make efforts to let children and their families, carers and guardians know we are thinkins of them and follow their journeys as much as possible. We try to offer emotional support and inforniative advice whenever we can. Many of the CureDM community face daily challenges such as the shock of diagnosis, illness, and often being in hospital. We hope, through our network, we can help support one another and let you know you are not alone. "CureDM Ims beell ll lifrline ofhope rea55urlluce tLI us. Getting the shock diugnosi.s, we didn't know where to turnfor infvrmahon ondfound.so little positive infvrmotion out there. The chority hns retslly helped our understonding and made usfeel more hopefvlfvr thefvture." Events of 2022 Families Day - Alton Towers - July We were able to organise an incredible farnily day to give families affected by CDM the opportunity lo enjoy the theme park with minin] cost lo them. This also gave families, carers, and guardians acrogs the UK the opportunity lo meet face-to-face and offer a safe space lo connect with other5 familiar with the IqSu of caring for chIldn a(lted by CDM. We received lots of lovely feedback aiK)Uthow much fun the allendees had and what a p0511ive experience il was. Cure If you attended Alton Towers this year you may have seen a friendly, somewhat cuddly character in the fonn of Miles the Mascot. We hope you enjoyed this new addition to our team. Watch out for him at OUT future eventsl "What afantostic eyeriencefvr our dllugliter to meet w so othcr children who slior condition. She's o met Iwo other clii who have thi5 condition... ren .I wish this charity had existed 11 yearb llgo when we collected herfrom hospitol...This chnrity is n tL*alkh of knowledge on tliis condition L7nd bringing u.4 together is uery.ywciul.
Disney On Ice - Sheffield, Birniinghant London - December We M'ere aIso able lo offer a Winter family event this year. We secured spacesal three venues, orfering55 people the chance to enjoy a fami]y trip to watch Disney on Icelive atBlrnYn, Sheffield, or London with Mini persona] cost to the fami]ies. Itwas lovelv to be able to give the13mi]ies another chance to mt up. For some itwas the first time attending one ol our evenLs. We hoFE those who could make it made some unforgettable memories for the festive seasonl It was SO successful that we are rekwating the offer in 2023- contact us if yoll areinterestedl "ICureDM}17As been constftnl Source o support LzKd knowledse (wer the pftst yellr wlien * tlw cliildren hllve bee ill, or I Imve hlld questions or ¢oncern.s. ** "Wg h&ve refilly LY7joyedand benefilledfrom thg two Meel ups (botli Alton Towers in Ilw Smmfrner und Disney on Ice in Deceillber) where ELiegol to Ineet Infiny olher fvMilie5 Wlth children wliohtsue Ilie saiiie condilioFI 115 Ply tlvo... .Thalfvr Ine is the best port os il I5 fillowed Iiie lo build a nettvoth rf support of oiher MHn4/dNds who begoiftg through similfjr...T1715 Ives me hove rcassmrunce, knu(ving l ftm Kol ftlolle! Research At last, 3potential 5y51emic treatments in clinical trials IAMO Pharni Avidity and Dyne) and more are on the way. CureDM are working with the companies to make sure the tria]s rneasurewhat is important to us, that they are not loo much ol a burdew and are fe. We are a150 workin8 with the UK resulatory b(Klies lospeed up the approval Pr(Ke and make them available to everyone as SlK>n as possible. Please get in touch if you'd like to know more or to Envolved! MDUK announ in 2022 that Dr.Chrig Tumer at UCL will leadll)g a nah31 '$10ry sdY ol adults with Con8enitsl DMI1sytnploms frotnbirthl. The SdY will sive us information to further llnprove care and a150 provide data for c]inical trials. MemEETS ol CureDM adv(rated for thi5 Study ard weare proud to i part-fundins it. We have been involved in pmducing a nurnlEr ol abstracts and Poste that have en preted atconferences by CureDM, TREAT-NMD, and the UK DM Patient Registrv Iwhich we also part-fundl to explain to cliniaans and researchers the real-world eyriences of the people that actually Iive with Myotonic Dystrophy. Thank you for the hundreds 0freSpo to ourongoing survey that allowed this lo haptEn. CU1111ndQIdh)0myOtnlrTrrOhVTyP)l7tr
Raising Awareness Shinxng a light on Myotonic Dystrophy especially the Congenital forni, As itucial. Here are some of examples how we spread the word this year: Celebratin8 International DM Awareness Day. ial media came into its own, esFecially Faceknk and'lwitter, where many in the UK communiiv and Eeyond showed support bv changing theirprolile pictur, sharing llrformation on the dise, u0m]ng triak, and wsting l)nal stories. The national mourningol the Queen meantlandmar that were planned to light up green on the litholseptember were purple re5FECt for the monarchy. Thei hai'e, however, all said thry11 light up lor usin 21 Growins Our Strons Online Presence: As well as the CureDM web site, this }'ear we added Instagram to ourcontinuous, strongoniine presence on sla1 mdi¥ helping us to share any news and breakthroughs. Ourprivate, UK only, Faceiwk gmp continued to grow. It is otds, for those lii'ingmth DM, locusingon peer supprl and day to dav living. Attending Conferences: This yeai we got back to face-t0-laceconferenc in the UK, Belgium Italy and Canada and al presented atthe pre5tigu5 IDMC. The5eallowed us to connect with the leAding rnedica] and ientilii prole55ionab, raisins the profile ol DM on a bigstage. Myoknnic TRERT-NMD tytropby Taking Part in Webinars: In 2022/20 we attended online conferaKegbad in theUK, acrogs Europe,Japan and the USA. As with -perSOn(onferenees, the webinats allow the exchange ol tnedicai adi'ances in the field of neuromuscular disease and create collaLx)ratiom thatrai* the prohle and accelerate awaren5 of Mj'otonic DtroPhY. Presenting the llnprtance ol patient prelerences webinars lor ISPOR IProle55ional &xich. lorHea]th Economics andoutcomes RarChI and reprnting thccornmunity in onlineconferences is vital lor keeping DM relevantin the eyes ol the reseaTchetS. Contactlng Professionals: We trreaSed eflorL4 to make positive contactwith medical professionals to bring recolOn and further their undetslandingol the disease, to speed up dia05183nd implement Ihellecessary care lo their patiellls living with Mvotonic Dystrophy earlier rather than later. Being tnetnbers ol Eurtr.mA (Federation ol European DM Ass(Klatiol and other DM SFecilic associationsenables us tobrirg meaningful di$$70n and collaboration to the very startolany proFct, making sure work is relevant lor the community werepresenl. Thi5year wecrealed a'could it be Mi'otonicDystrophy. ter that ha5been distributed to clinican5 and sent aroundthe world. This can ir dowtdoaded Irotn our website OT please COntt us Iorcopies. Myoionlc wwM'.iureilm.co.uk IDMC cortirn Awarene55 D tembei LDITtsEMY DY TA Raising Funds! None of this would hap1 without the generous organtsations that provide us with grants and, ofcourse, the ma amazing people that volunteer for us and selflessl}' raise the much needed moneyl You are fabulousl fkLV( FUTrX> For Inore illfvrFnfjfrion on CureDM and what we are doing beliiiid flye 5cene5, you keey up lo dufe on OUT SO(ial media page5, Visil our webstle, or e-ttmil us al curedm@ouilook.com Cure Mytstonic Dystrophy UK Charity wwwLuredm.co.uk Reg.No.I191217 INDRAISING GULATOR Page4