Welcome to Duchenneber, a twenty-four day campaign throughout December, to raise awareness and funds for Duchenne Muscular Dystrophy. Check back here each day and open up a new door for updates on research, patient stories and news about exciting projects at Duchenne UK, our partner charities, family funds and much more.
Today we are launching Duchenneber: a twenty-four day campaign to raise awareness and funds for research into Duchenne Muscular Dystrophy, in collaboration with our new charity partner, Duchenne Now.
To celebrate us coming together for the common cause of accelerating the search for treatments, we want the world to hear about our incredible sons, daughters, children, teenagers and adults, all bravely fighting Duchenne Muscular Dystrophy. And pay tribute to their incredible families.
Building on the success of Duchenne World Awareness Day, we want to dedicate this month to spreading the word about Duchenne Muscular Dystrophy, the ways in which the research landscape has changed, and what we are doing to help accelerate the search for treatments and a cure.
We are entering exciting clinical times for this disease. Now more than ever, we need to raise the profile and awareness of Duchenne Muscular Dystrophy.
Do please visit this website every day to see what’s new. Please SHARE Duchenneber – we want to get it out there. And if you have ideas of how we can improve Duchenneber for next year then do please let us know.
This month we are going to be sharing updates from the community, updates on research projects, details of what we have been working on over the year, and some new announcements.
Thank you to everyone who has joined us in our fight to End Duchenne. Together we are stronger. Together we will make history.
Click the link below to read our exciting news about gene therapy treatments!Exciting gene therapy news!
Duchenneber is a symbol of our recent collaboration with Duchenne Now. We would like to welcome any new Duchenne UK supporters who have come to us from Duchenne Now.
Duchenne Now was founded in 2012, by parents of children and young adults with Duchenne muscular dystrophy, who between them have more than 20 years of dedicated Duchenne charity experience. Their charity was born out of their desire to ensure transparency, accountability and an ambition that all funded projects have a clear road map to market.
Duchenne Now and Duchenne UK share the same dedication in our joint mission to fund and accelerate the search for treatments and a cure for Duchenne for all.
At Duchenne UK we are delighted that Duchenne Now have joined forces with us. Founders of Duchenne Now, Vici Richardson and Tony Levene, have been running the charity since 2012.
Vici Richardson, founder and trustee of Duchenne Now said:
"Over the last five years I am proud to say with our members support we have contributed over half a million across many projects with more still in the pipeline. On top of that we have ensured a shift in how charities in our community are working. Joining forces with Duchenne UK is the next logical step- to share knowledge, our advocacy experience, our funds and expertise to accelerate the fight for a cure.
"Merging Duchenne Now with Duchenne UK will be a great thing for our community. I value the friendship of the founders as well as the tireless work they do. I'm not giving up, far from it, never in the 14 years [since my sons diagnosis] have i felt so much hope for a treatment for Duchenne. But the time has come to take a step back and support those who will take this on."
Tony Levene, founder and trustee of Duchenne Now said:
"We've been working alongside Alex and Emily from Duchenne UK for many years, and I am blown away by the progress they have made. They have attended hundreds of meetings, and have put many quality projects together that Duchenne Now and others have supported. I feel that is it in the best interests of the community for us to join forces, because together we will be stronger."
We are looking forward to the new year, where we will be having discussions with Duchenne Now about choosing which research projects to fund.
It would be great if you could use this opportunity to sign up to regular giving to Duchenne UK.Sign up to Duchenne Direct
Duchenne UK is hosting two more Patient Information Days. These will take place at UCL Great Ormond Street Institute of Child Health in London and at the Queen Elizabeth University Hospital in Glasgow.
Patient information days are primarily aimed at educating parents and caregivers about:
There will also be the opportunity to receive the latest care updates, and learn more about supporting the learning and education of children and adults living with Duchenne muscular dystrophy.
REGISTER HERE NOW TO ATTEND: https://duchenneuk.eventbrite.com
Registration will open in January for the Glasgow patient information day. Email us at firstname.lastname@example.org if you have any queries.
We couldn't have achieved what we have without the support of our wonderful patrons.
Krishnan Guru-Murthy came up with the idea of a bike ride from London to Paris in 24 hours. We called it the Duchenne Dash.
Last year the Dash raised a staggering £1million. In total, all 5 rides have raised over £2.5million!
Thank you Krish!
Last year, Duchenne UK commissioned Professor Steve Winder at Sheffield University to investigate a nutraceutical soy product, called Haelan 951.
Haelan 951 has been popular with some DMD families as a perceived way of slowing down disease progression. It has a very unpleasant taste and is expensive. It has never been tested in clinical trials, and the only evidence for its effectiveness is anecdotal.
Prof. Steve Winder carried out a study in mice which looked at the various properties in Haelan 951. He then divided them up into different diets, and compared them to each other and to a placebo group.
The results clearly showed that one of the diets had a significant and positive effect on the mice.
Duchenne UK has now asked Professor Winder to validate and extend these findings in a new set of experiments.
We will keep you updated with his progress.Take a look at the results here!!
Today, Emily Crossley, the co-founder of Duchenne UK, is chairing a Duchenne Muscular Dystrophy Awareness Day. This is a conference aimed at investors. Speaking at the conference alongside Emily, will be some of the leading professionals in this field.
Professor Dame Kay Davies FRS, from the University of Oxford, will be providing an overview of Duchenne muscular dystrophy: exploring molecular and cellular pathologies and possible therapeutic strategies. Rosaline Quinlivan MD, from the Dubowitz Neuromuscular Centre at Great Ormond Street Hospital (GOSH), will be covering the clinical presentation of DMD across all age groups. She will also be discussing the merits of different types of intervention at these stages. Professor Thomas Voit, from the UCL Great Ormond Street Institute of Health, will be talking about the current drug candidates in the pipeline. He will be discussing outcomes of trials, strengths and challenges and evolution in relation to a changing regulatory landscape.
Later, there will be company presentations for the emerging pipeline of DMD therapies, and an opportunity for Q&A. There will be presentations from Summit, Santhera and Catabasis.
This unique event is bringing together clinical practitioners, biotech and pharmaceutical industry professionals, patient groups, media and speciality investment professionals to discuss DMD, developments in the space, and share experience and information.
The event is from 10am-1pm and will be live streamed.Watch a live stream of the event here
Duchenne muscular dystrophy (DMD) was first described in 1830. We are now in the Golden age for DMD research.
Look at our timeline of the history of Duchenne muscular dystrophy to see how far we have come!
Take at look at Our Impact on the Duchenne UK website to see what we have achieved with the help of donors, partner charities and researchers.OUR IMPACT
We are a lean, ambitious charity. We work hard and keep our costs low to ensure as much of the money you raise goes towards finding a cure for Duchenne. Head over to OUR MISSION on the Duchenne UK website to hear what your money could do to help.
There are many ways you can help Duchenne UK. Any donation, big or small, goes towards helping find new treatments and a cure for Duchenne.
Help us End Duchenne.
You can do anything to fundraise for Duchenne UK. We have come up with some ideas of things you could do to raise money. But you can use your imagination!
Head over to Duchenne UK's Virgin Money Giving page and click 'Start Fundraising' today.Start fundraising for Duchenne UK
Duchenne UK is proud to be funding Decipha, an educational support programme that is free for families. Decipha was set up by Janet Hoskin and Nick Catlin. The Decipha team work face to face with families and offer a range of assessments and valuable support to help them navigate educational changes. This might involve training at school or some advice or support over the phone, or a full assessment followed up by detailed reports and reccommentdations towards Educational Health and Care Plans. This is a FREE service to DMD families.
Kate Tinkler's son Mitchell has Duchenne muscular dystrophy. She recently received support form Decipha and has written a short blog about her experience. Click the link below to read her blog and some more information about Decipha, from founder Nick Catlin.Click here!
Alex's Wish has been a partner charity with Duchenne UK since 2016. We are so pleased to be working closely with the charity.
Here are a few words from Emma Hallam, Alex's Mum and the founder of Alex's Wish.
"Alex’s Wish is a charity based in the East Midlands who are 100% focussed on bringing new treatments – and ultimately a cure to all boys living with Duchenne. Our supporters have been amazing this year so we have been able to put £135K into promising projects to help all boys – including a Tamoxifen trial, gene therapy, repurposing projects and helping to expand clinical resource in the UK. Having organised a range of community and fundraising events throughout the year we now have over 1,000 supporters giving to our cause, and this is growing year on year.
We are equally excited about the year ahead, 2018 is going to be a year of growth for us and we hope to be able to give a further £150K+ to Duchenne UK. In 2018, we will be launching our Business Club, with an aim of raising a minimum of £25,000 per year – this will help us engage with local businesses across the East Midlands to give regularly ensuring we can continue to raise a stable income for years to come. We are a lean, committed and passionate charity with little overheads and focussed on working solely with Duchenne UK as a Charity Partner. Together we are so much stronger and will a much better chance of making a real difference to boys with Duchenne."
To learn more about what Duchenne UK has done with the support of our partner charities, read our impact below.OUR IMPACT
We would like to say a huge thank you to our family and friends funds for all the fantastic fundraising they have done throughout 2017.
Our Family and Friends Funds provide vital support to Duchenne UK by fundraising for us and helping us to advocate to accelerate research. They share our ambition to fund the most promising research, no matter where it is in the world, and to do so with a vision to help bring that research out of the laboratory and into the clinic where it can help patients.
Click the button below to take a look at some of the fantastic work our family and friends funds have done this year.CLICK HERE
The support we receive from SCHOOLS and BUSINESSES is an invaluable part of our fundraising. There are many ways we can work with schools a businesses and can offer lots in return to firms and schools who want to make us their Charity of the Year!
Duchenne UK is a lean, ambitious charity with a clear vision: to fund and accelerate treatments and find a cure for Duchenne Muscular Dystrophy (DMD). In the last 5 years Duchenne UK, in collaboration with Joining Jack, has raised over £10 million. In that time £6.5 million has been spent on research into finding a cure for Duchenne muscular dystrophy, a further £3.5 has already been committed to future projects. We keep our overheads low, so every penny we raise counts.
You can help us to end Duchenne by choosing us as your charity of the year.
Duchenne UK was Compass and Foodbuy's Charity of the Year 2017. Foodbuy had a team take part in the Duchenne Dash 2017- a single cycling event which raised over £1 million. Managing Director of Foodbuy, Oliver Cock said:
"We've worked closely with Duchenne UK this year and being a smaller charity, led by two inspiring founders, it's been much easier to establish a meaningful connection. Our employees feel part of something tangible and important, and have raised more money than they did for more established charities. Their Duchenne events, including the Duchenne Dash, are hugely life-affirming, inspiring, meticulously organised and original."
If you are interested in making us your Charity of the Year, email us at email@example.com.
Read Our Mission to see how we plan to end Duchenne.Our Mission
We couldn't have achieved what we have without the support of our wonderful patrons.
Owen has been fantastic at helping us promote awareness for DMD, with his signature 'JJ Salute' when he scores.
THANK YOU OWEN!
Today marks the publication of Janet Hoskin's book: 'A Guide to Duchenne Muscular Dystrophy: Information and Advice for Teachers and Parents'
Janet currently teaches on the undergraduate and postgraduate Special Education programmes at the University of East London and is involved in several research projects that focus on young people with SEND and life limiting impairments.
Janet has worked on various projects for yound people with DMD, she has also worked with children and young adults with a range of Special Educational Needs and Disability though Decipha CIC, in particular she led a Department for Education funded Leadership Project for young adults with life limiting impairments from 2012-2014. Duchenne UK fund Decipha CIC to work with young people with DMD and their schools to support any barriers to learning they may face. If you are worried about issues concerning school, contact Decipha.
'A Guide to Duchenne Muscular Dystrophy: Information and Advice for Teachers and Parents' brings together chapters by a range of experts in DMD aimed at supporting young people and their families as they move through school and college. It is not primarily aimed at the physical progression of DMD but rather focuses on learning and behaviour challenges. Chapters include advice on assessment, reading, behaviour strategies and planning as well as how to talk to children about having DMD. Most of all it's about getting the best life possible if you have Duchenne Muscular Dystrophy.
Emily Crossley from Duchenne UK said:
"This book is long overdue and will be a welcome resource for parents, caregivers, teachers and patients with Duchenne muscular dystrophy. It provides a compass with which to navigate the world of Duchenne muscular dystrophy, giving patients and caregivers they need to manage the emotional, behavioural and learning challenges that sit alongside the physical realities of DMD. I found the chapter on talking to your children especially helpful, as I am very often asked by families when is the "right time" to tell their children. This book shows that although there is no right time, there are ways of addressing these unbelievably difficult conversations that will help. And there is lots else in the book to give parents the hope that their children can shoot for the stars and create as Dr Jon Hastie so movingly described a "fantastic life" "
Janet Hoskin has recently become a member of the Duchenne UK Patient Advisory Board. Our Patient Advisory Board (PAB) is made up of parents of children and young adults of different ages, who advise and give feedback on the projects we are considering for funding.
If you would like to come to the launch of Janet's book, register for our Patient Information Day at GOSH in February here.
If anyone would like a FREE copy of Janet's book, sign up for the Duchenne UK newsletter and email us at firstname.lastname@example.orgPatient Information Day
DMD Pathfinders is a user-led organisation: run by adults with DMD for adults with DMD. DMD Pathfinders provides the unique perspective of adults who have lived with the condition all their lives. As well as providing advice and information, DMD Pathfinders are determined to ensure adults are not left behind in the race for a cure.
Jon Hastie, CEO of DMD Pathfinders, shares what he has been doing with Duchenne UK to promote research and treatments for adults living with DMD.Read Jons' Blog
What a year we have had at Duchenne UK!
We have been incredibly busy in our mission to end Duchenne.
Read our end of 2017 round up to see where we have got to in our mission.
Duchenne UK 2017 round up!
The global preference survey is a research survey on preferences for treatments for Duchenne muscular dystrophy (DMD): for individuals aged 18 and up with DMD and/or parents or guardians of children with DMD.
Biopharmaceutical companies that are working to develop new drugs for DMD are interested in the treatment priorities of the DMD community. DMD advocacy organizations internationally have come together to launch a new survey to help answer these questions so that patient and caregiver preferences can better inform drug development and approval. This research survey is online and anonymous, meaning that no one knows who took the survey or what they said.
The goal of the survey is to learn about your priorities and preferences about treatments for DMD.
The survey will provide important information. This is a critical time for the voice of people affected by DMD to be heard because the results of this research will be used to inform policy and drug development. We hope you will consider participating. There is no requirement to participate. We are committed to sharing the information we learn from the survey with our DMD community.
The survey is launching in January and we need 60 families to complete the survey. If you would like to help by completing the survey please email: email@example.com.
In the summer of 2017 Duchenne UK launched its first patient and caregiver survey. If you would like to know more about the results of that survey, watch the video below.
Duchenne affects approximates 1 in every 3,500 boys that are born but only around 1 in every 50 million girls. It may be rare, but it does happen. We need to raise awareness for girls living with Duchenne.
We have been speaking to Feriel, a 26 year old woman living with Duchenne muscular dystrophy. She has written us a short blog about her experiences with Duchenne from diagnosis to now.
Follow the link below to read more about girls with Duchenne and to read Feriel's blog.read more
We would like to congratulate the Joining Jack team for their recent victory! Joining Jack took a team to the Dubai 7's international rugby competition, and they came back champions!
Joining Jack is a founding charity partner of Duchenne UK. Joining Jack and the Duchenne Children’s Trust came together in 2016 to form Duchenne UK, a charity dedicated to funding and accelerating research and treatments solely for Duchenne muscular dystrophy (DMD).
Alex Johnson, founder of Joining Jack and Jacks Mum, tells us more about Joining Jack's victory at the Dubai 7s.Read here
Duchenne UK is a lean, ambitious and highly focused charity with a clear vision to fund and accelerate treatments and a cure for Duchenne Muscular Dystrophy (DMD), and to do so in a decade.
In 5 years, Duchenne UK has raised more than £10 million. We've funded more than 50 research projects to find treatments and a cure for Duchenne Muscular Dystrophy. We helped change regulation to speed up access to promising new therapies. We've funded pre-clinical work on groundbreaking gene therapy which is now being tested on boys with Duchenne.
The DMD Hub was created by Duchenne UK and leading neuromuscular clinicians based at the John Walton Muscular Dystrophy Research Centre in Newcastle and Great Ormond Street Hospital in London. The DMD-Hub is addressing the lack of capacity in the UK for clinical trials in Duchenne Muscular Dystrophy. Our ambition is to ensure that every child diagnosed with DMD in the UK is given the opportunity to take part in research.
During the first year of operation the DMD Hub has successfully achieved, even surpassed, the immediate priorities identified. Read the article below to see the first year progress of the DMD Hub.
Robyn is a mother to Felix, one of the World’s Strongest Boys.
Felix has Duchenne Muscular Dystrophy. We believe that living with Duchenne makes our children anything but weak. It makes them incredibly strong. We call them the WORLD’S STRONGEST BOYS. And with your help, we can save them.
In Robyn’s video, she discusses her son's diagnosis and the impact of the disease on their family life. It's a devastating situation but, rather than just accept it, Robyn, her son and her whole family resolved to fight it.
More about the World's Strongest Boys
This year Duchenne UK were donated over 200 Christmas gifts through Heart Radio's Giving Tree campaign. The campaign, run at Bluewater shopping centre, gives Christmas shoppers a chance to buy an extra present to give to a disadvantaged child.
We would like to thank Becky Burnett, one of our amazing volunteers and a member of our patient advisory board. Becky, along with some friends and some of the Duchenne UK team, spent Tuesday wrapping presents and sending them to children at DMD Hub sites who are undergoing clinical trials.
We would like to thank Becky, Heart Radio and Bluewater shopping centre.
You have made it to the end of Duchenneber 2017! We hope you have enjoyed the first ever Duchenneber. If you have any suggestions for next year please let us know!
Thank you all for your endless support.
Seasons greetings!Donate to Duchenne UK