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.

Happy Duchenneber!

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An Introduction from Alex and Emily

December 01

Welcome back to Duchenneber  - a whole month dedicated to raising awareness and funds for research into Duchenne Muscular Dystrophy.

We are in exciting clinical times for this disease. Now more than ever, we need to raise the profile and awareness of Duchenne Muscular Dystrophy.

Each day on our DUCHENNEBER ADVENT CALENDAR we will feature news about research projects, new resources for the community, videos & podcasts.   

Thank you to everyone who has joined us in our fight to End Duchenne. Together we are stronger. Together we will make history.

We couldn’t have done it without our incredible supporters. Thank you all in this festive month, for everything you have done and continue to do to help us. 

Please share DUCHENNEBER, change your profile and spread the news so that we can reach the widest audience possible. 

Happy DUCHENNEBER!

Alex, Emily and the Duchenne UK team


Number of children on clinical trials doubles at Great Ormond Street Hospital, following funding through the DMD Hub

December 02

Duchenne UK is pleased to share a report from Great Ormond Street about the positive impact of the clinical staff posts we fund at the hospital.

View the GOSH report here

A new podcast brought to you by Duchenne UK

December 03

We are thrilled to be launching our new podcast series DECODING DUCHENNE

In the first episode of our podcast series, our host Clare Runacres talks to Emily Crossley and Alex Johnson about why they co-founded Duchenne UK. They discuss the work the charity is doing to look at how where ever possible, we can accelerate DMD research and work with industry and regulators to get drugs approved.

Listen here:

SUBSCRIBE HERE

Standards of Care and the New DMD Family Guide

December 04

Duchenne UK want to ensure that everyone living with Duchenne has access to the best possible care, therefore we strongly encourage all parents, caregivers and patients to read the DMD Family Guide and the updates to the standards of care, which reflect the significant advances in Duchenne research.

Please CLICK HERE to view the New 2018 DMD Guide for Families - UK Version.

Earlier this year, Lisa Kuhwald attended the Duchenne Caregivers’ Masterclass in Madrid on behalf of Duchenne UK, as part of her voluntary role as our Advocacy Support Officer.

The masterclass focussed on the newly published standards of care for DMD. Lisa wrote a blog which summarises what she took, as a parent, from this meeting: 

Read Lisa's Blog Here

REGISTRATION IS NOW OPEN!

December 05

We our hosting our fourth FREE Parent and Caregiver Information Day, on 16th March 2019 at Guy’s Hospital in London. 

The focus of the day will be on gene therapy. It will provide parents and caregivers with updates on DMD research, clinical trials and care.

Read about our previous Information Days HERE.

REGISTER FOR YOUR PLACE NOW

Decipha: RoadMap for Life

December 06

Duchenne UK is proud to fund Decipha, an organisation which was set up by Nick Catlin and Dr Janet Hoskin to benefit families, young people and adults with special and additional needs. We are pleased to say that more than 100 families have been helped, with advice on education and assessments.

In the past 2 years we have awarded £60,000 to Decipha to fund their ‘RoadMap for Life programme’, which provides person centred planning, assessments, reports and training to identify key outcomes required for Education Health and Care Plans for young people living with Duchenne muscular dystrophy.

Nick from Decipha spoke to us about the RoadMap for Life programme:

“Decipha, through its partnership with Duchenne UK, provides a unique service to families and professionals supporting boys living with Duchenne Muscular Dystrophy. It provides expert assessment and evaluations based upon agreed international standards of psycho-social care and educational research. We take that knowledge and experience and share this with families and schools to ensure we can get the best possible outcomes for Education, Health, Care and Life. 

Over our two-year partnership, we have worked with over 100 families offering advice, support, assessments and advocacy.

We have worked with 22 SENCO's in schools and many other teachers and TA's. We have delivered workshops for parents and in school staff training.

Duchenne UK has offered invaluable funding, expertise and encouragement to the RoadMap for Life project that is now having a real impact on improving the quality of life of young people and adults living with Duchenne Muscular Dystrophy.”

We would like to thank the team at Decipha for their hard work.

Dr Janet Hoskin edited and released a book titled "A Guide to Duchenne Muscular Dystrophy: Information and Advice for Teachers and Parents”. Duchenne UK are offering this valuable resource free of charge to UK families. To claim your free copy, email us at info@duchennuk.org

Please take a look at the Decipha website and reach out them if you need any educational support. 

Get in touch with Decipha here.

Family and Friends Funds

December 07

Today we want to give a huge and heartfelt thanks to our Family and Friend’s Funds for their continued support and hard work for Duchenne UK. Together they raised a staggering £600,000 in 2018 to help us in our fight to end Duchenne!!

THANK YOU

We know that the people who are most passionate about raising money to find treatments and a cure for Duchenne muscular dystrophy are those who live with it each and every day. 

We set up our Family and Friends Funds to support the friends and families of those living with DMD to raise the money to accelerate treatments and to find a cure. It's a race against time to give those with DMD a different future, but by working together we are hopeful we can do just that.

Our Family and Friends Funds fundraise for us and help 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.

Please watch this film about our funds:


Find out more here

Joining Jack: Dubai 7's Tournament

December 08

Joining Jack is a founding charity partner of Duchenne UK. In 2016, Joining Jack and the Duchenne Children’s Trust came together we to form Duchenne UK, a charity dedicated to funding and accelerating research and treatments solely for Duchenne muscular dystrophy (DMD).

Joining Jack was set up by Alex and Andy Johnson after their son Jack was diagnosed with DMD. Andy Johnson is an ex-professional rugby league player and the rugby community have long supported Joining Jack. 

For 7 consecutive years the 'Joining Jack Legends' team have entered the Emirates Dubai Rugby 7's Tournament. Each year the team has been made up of rugby legends, who all take part to support Joining Jack and raise awareness of Duchenne muscular dystrophy.

A message from Alex, co-founder of Duchenne UK and founder of Joining Jack:

"Why take a team of ex-professional rugby players to the Dubai Sevens?

The answer is quite simple - to raise awareness of Joining Jack and Duchenne Muscular Dystrophy.

How does raising awareness help to find a cure for disease?

When Jack was diagnosed with Duchenne seven years ago it was Andy’s ex-team mates who immediately answered our call to action to help find a cure for Duchenne.

They wanted to know what they could do to best help us. Andy’s answer was simple, help us to get people talking about Duchenne.

We realised the more awareness there is of Duchenne, the more private and public money will be put into funding research to help us find a much-needed cure. It will also mean more researchers and companies would also be attracted to come and work in the Duchenne field.     

Our ambassadors have taken Duchenne to their hearts and now use their positions to talk about Duchenne at every opportunity. At the Dubai Sevens this year Andrew Farrell recorded a podcast about Joining Jack that will be available to listen to on all Emirates airlines flights. This will raise awareness of our charity and Duchenne around the world.

Joining Jack Legends have helped us raise hundreds of thousands of pounds through sponsorship, runs, rides, gameshows and dinners.

We are grateful to our ambassadors and the Joining Jack legends for helping us bring Duchenne to a bigger audience.

We would not be in the position we are today of emerging treatments without the support of the rugby community."



Visit the Joining Jack website

Owen Farrell and Andy Farrell OBE

December 09

We would like to dedicate today to thanking two of our wonderful patrons, Owen and Andy Farrell. 

Owen Farrell is an English professional rugby union player, currently playing for Premiership side Saracens. Owen has played international rugby since 2012 and became the Captain of the England team this year. He has been part of helping raise awareness for DMD since our co-founder Alex’s son Jack’s diagnosis. Owen’s signature celebration is the JJ (Joining Jack) salute. 

"My dad and Jack’s dad played together at Wigan and our families are very close," says Owen. "The charity has been a huge part of our family’s lives. Everyone who donates or who tells their friends and they then go on to spread awareness or raise money, then they are helping. If I can help spread awareness by doing the salute when I kick a goal to do this, it’s the least I can do."

Owen’s Dad, Andy Farrell OBE, is an English rugby union coach and former rugby league and rugby union player. He is currently the defence coach for Ireland and is set to become the head coach after the 2019 Rugby World Cup. 

Andy Farrell: "I feel honoured to be an Ambassador for Duchenne UK; a charity set up to help raise much-needed awareness of the fatal disease Duchenne muscular dystrophy. When Andy Johnson (of Joining Jack) asked me to help the first thing that came to mind was my own two sons, as this mainly affects boys, and if anything was ever to happen to them I would hope and pray that I would also get support from friends. AJ has been a great friend since we started to play rugby together and to help him with this charity is a great privilege."

Owen and Andy provide a huge amount of support for the charity. They were both heavily involved in our Rugby Dinner in September, which raised £400,000 to go towards our mission to end Duchenne. Andy also played in the Joining Jack team in the Dubai 7's tournament last week. 

Keep your eyes peeled for tomorrows Duchenneber post, which contains an interview with Andy Farrell himself. 


NEW PODCAST EPISODE DECODING DUCHENNE

December 10

In this weeks episode of Duchenne UK's DECODING DUCHENNE podcast series, our host Clare Runacres interviews Andy Farrell OBE about his involvement with the charity.

To listen to the first two episodes follow the link to subscribe below or search "Decoding Duchenne" in your Podcasts app.

This is a really exciting new way to help raise awareness for Duchenne UK and Duchenne muscular dystrophy so please SUBSCRIBE on iTunes, rate the podcast and tell ALL your friends to listen too!

SUBSCRIBE HERE

How to use the DMD Hub Clinical Trial Finder

December 11

We developed the Clinical Trial finder on the DMD Hub website which has:
• Every existing and pending clinical trial for Duchenne in the UK
• Clear and detailed summaries of each trial so you can make an informed choice
• Trustworthy and up-to-date information

Please watch and share our Clinical Trial Finder tutorial video to learn how easy it is to access information about clinical trials for DMD on the DMD Hub website:

Join the Hub to stay informed about the latest trials.

GO TO THE CLINICAL TRIAL FINDER

Please complete our survey

December 12

Duchenne UK is testing a number of what are called repurposed medicines to see if they are effective in DMD. Repurposed medicines are medicines that are already approved for use in one condition, which may show promise in other diseases. Please read about one example, the Tamoxifen trial, Duchenne UK is co-funding.

We would like to invite parents/caregivers and patients with DMD to complete our survey to help us better understand how medicines (prescription and non-prescription) and nutraceuticals are currently being used by those living with Duchenne muscular dystrophy (DMD). 

We require some feedback from the Duchenne Community that will help us understand: -

  1. What prescription medications are taken by those living with DMD.
  2. To what extent patients are sourcing their own prescription medications (un-prescribed or off-label).
  3. What nutraceuticals are taken by those living with DMD.

The results of the survey will help Duchenne UK make informed decisions about the development of repurposed drugs as potential treatments for DMD and will facilitate clinical trial planning.

They survey is completely anonymous and will not ask you for any personal details.

The survey can be filled in via your mobile, PC or Laptop and will take no more than 20 minutes to complete.

You can complete the survey by clicking the link below:

https://www.surveymonkey.co.uk/r/YDXV37P

If you are a caregiver for more than one child/adult living with DMD please complete one for each child/adult as experiences for each may be different for each.

The survey will close at midnight on Tuesday 2nd January 2018.

Duchenne UK would like to thank you in advance for providing such valuable information and helping us move a step forward in increasing our knowledge in such an important area within Duchenne research.

For more information please contact research@duchenneuk.org

COMPLETE THE SURVEY HERE

Tamoxifen Open Label Extension Study

December 13

In July 2017 we announced €675,000 funding towards TAMDMD, a phase 3 clinical trial to test the breast cancer drug tamoxifen for use in patients with Duchenne muscular dystrophy. This funding provides the research team with a project manager as well as funding a site in Europe and two sites in the UK, Alder Hey and Leeds.

In June 2018 the first patients were dosed in this trial. So far, 12 patients have been dosed from a total of 99 participants. 

But what happens when the trial is over? We think it is important that all trial participants (especially those being on the placebo arm) have access to the treatment once they have completed the trial. This is the case in most clinical trials and is known as the Open Label Extension Study.

That is why we are committing an additional £780,000 to the TAMDMD trial, for an open label extension study. The additional investment brings Duchenne UK’s total commitment to the Tamoxifen trial to just under £1.4million.

The open label extension study will allow participants to continue in the study after they have completed the main trial.  During this extension study all participants will be taking tamoxifen, whereas in the main trial half of the participants will have been taking a placebo. Efficacy data will still be collected and participants and safety monitoring will continue throughout.

The open-label extension study will help gather long term data on the effects of tamoxifen on patients with DMD.

Duchenne UK would like to thank our partner charities and funds for their support of this project: Alex’s Wish, Duchenne Now, Joining Jack, Archie’s March, Help Harry, Jacobi’s Wish, Jack’s Aim, Team Felix and The Smedley Family.

Read the full press release here

Wheels of Change: BUILDING THE DREAM CHAIR

December 14

We partnered with Whizz-Kidz and the University of Edinburgh to win £1 million from the People’s Postcode Lottery Dream Fund to develop a dream chair for wheelchair users.

Whizz-Kidz have sent us an update on the project, read it here. 

One of Duchenne UK’s own ambassadors, Saul, featured in Whizz-Kidz video to Peoples Postcode Lottery, which helped secure the £1 million grant.


Read the Whizz-Kidz update here.

DMD Pathfinders

December 15

DMD Pathfinders is a user led charity which promotes choice, control and quality of life for teenagers and adults with Duchenne Muscular Dystrophy in the UK. It campaigns for improved standards of health and social care and provides advice, guidance and support to teenagers and adults with Duchenne muscular dystrophy on issues such as independent living, housing, employment and welfare rights.

DMD Pathfinders helps teenagers and adults with DMD life longer, happier lives.

Please contact DMD Pathfinders if you need advice.

Jon Rey-Hastie is the CEO and one of the co-founders of DMD Pathfinders. Jon Completed his PhD in 2008 and has since worked for several disability charities and as a local government officer. Jon enjoys gaming, travel and cocktails. Jon has Duchenne muscular dystrophy. 

Duchenne UK work closely with DMD Pathfinders. In October, we co-hosted an information day for Adults with Duchenne, through the DMD Hub. Amongst a rapidly developing research pipeline of new treatments for DMD, how can we ensure these treatments benefit adults?

Jon summarised the Adult DMD Hub day on the DMD Pathfinders website. 

Jon attended Duchenne UK’s Rugby Dinner in September, where we raised £400,000. Jon wrote a powerful blog, which touched on the current landscape of DMD research and the strength of the DMD community. Take a moment to read Jon’s blog.

Last year, Jon wrote a piece for Duchenneber about titled: ‘Life as a Disabled Man: What is Normal Anyway?’. 

DUCHENNE DASH

December 16

In June, 160 riders embarked on the challenge of cycling from London to Paris in 24 hours in our biggest annual fundraiser- the DUCHENNE DASH.

Together, they raised more than £1 million for Duchenne UK. This money will help us in our mission to END DUCHENNE.

We would like to thank our incredible riders and sponsors, and all the generous donors who helped us reach this incredible total.




NEW PODCAST EPISODE DECODING DUCHENNE

December 17

In this weeks episode of Duchenne UK's DECODING DUCHENNE podcast series, our host Clare Runacres speaks to Emily Crossley, Fleur Chandler and Josie Godfrey about Project HERCULES.

Project HERCULES is a collaborative global project set up by Duchenne UK to increase the chances of patients with Duchenne Muscular Dystrophy (DMD) of accessing innovative treatments. It brings together pharmaceutical companies to develop and build a better evidence base to help support their case for the pricing and reimbursement of new treatments for DMD. 

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To listen to the first three episodes follow the link to subscribe below or search "Decoding Duchenne" in your Podcasts app.

This is a really exciting new way to help raise awareness for Duchenne UK and Duchenne muscular dystrophy so please SUBSCRIBE on iTunes, rate the podcast and tell ALL your friends to listen too!

SUBSCRIBE HERE

A film about some of the challenges in gene therapy and a promising new approach

December 18

2018 has been a big year for Duchenne UK and the Duchenne community. We have commissioned novel research, funded clinical trials and helped hospitals run those trials by funding doctors, nurses and support staff. And we are leading a ground-breaking collaboration with pharmaceuticals companies to get treatments to patients faster. 

Part of Duchenne UK’s mission is to make sure we find and fund projects that cover all areas of research that demonstrate a potential to treat and cure DMD. We want to leave no stone unturned!

One of the projects we have announced this year is a grant of £655,000 to Evox Therapeutics to investigate an exciting new way of delivering gene therapy to patients.

Please watch our film which explains more about gene therapy, Evox Therapeutics, the discovery of exosomes and why this project is important and exciting:


READ MORE ABOUT THIS PROJECT

Eight pharmaceutical industry leaders join Phase 2 of Project HERCULES

December 19

Duchenne UK are delighted to announce that eight industry partners are supporting Phase 2 of Project HERCULES in 2019.

Catabasis Pharmaceuticals, Pfizer, PTC Therapeutics International Ltd, Roche, Sarepta Therapeutics, Solid Biosciences LLC, Santhera Pharmaceuticals Holding AG and Wave Life Sciences USA, Inc will join Duchenne UK and other partners in this ground breaking global project.

Project HERCULES will develop a disease level economic model and other tools to better demonstrate the real value of new treatments for DMD for Health Technology Assessments and reimbursement decisions.

Read the full press release here.

Bristol Royal Hospital for Children Joins the DMD Hub as a Hub Site

December 20

As part of our commitment to expanding clinical trial capacity throughout the UK, we are delighted to announce that Bristol Royal Hospital for Children is joining the DMD Hub as a Hub site. 

Duchenne UK has awarded £41,151 to the trust to recruit a Clinical Trial Coordinator. This funding is part of Duchenne UK and the DMD Hub’s mission to increase clinical trial capacity in the UK. 

Read more on the DMD Hub website.

Thank you to all of our incredible fundraisers

December 21

We would like to say a special THANK YOU to every single one of our fantastic fundraisers.

The funds you raise enable us to fund the most promising research and continue on our mission to END DUCHENNE. 

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If you want to get involved next year, there are many ways you can fundraise for Duchenne UK and help us accelerate research to find treatments and a cure for DMD. Please email events@duchenneuk.org for fundraising advice or to register your interest in an event for Duchenne UK

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We have places in sporting events throughout 2019, including:

Wigan 10k and Family Mile - this event is run by our partner charity Joining Jack

Greater Manchester Marathon- please get in touch before the end of January to secure your place!

Duchenne Dash 2019

Cheshire Dash 2019

Wigan Bike Ride

Tough Rugby Tournament

Prudential Ride London-Surrey 100

Great North Run

Manchester Half 2019

Royal Parks Half 2019

Tri4Duchenne SAVE THE DATE 15th September 2019. More information on this event will be released in the New Year. 


Visit our website to see how else you can fundraise for Duchenne UK.


More information about upcoming events.

Repurposing Survey closing 2nd January- please help us now

December 22

Duchenne UK is testing a number of what are called repurposed medicines to see if they are effective in DMD. Repurposed medicines are medicines that are already approved for use in one condition, which may show promise in other diseases. Please read about one example, the Tamoxifen trial, Duchenne UK is co-funding.

We would like to invite parents/caregivers and patients with DMD to complete our survey to help us better understand how medicines (prescription and non-prescription) and nutraceuticals are currently being used by those living with Duchenne muscular dystrophy (DMD). 

We require some feedback from the Duchenne Community that will help us understand: 

  1. What prescription medications are taken by those living with DMD.
  2. To what extent patients are sourcing their own prescription medications (un-prescribed or off-label).
  3. What nutraceuticals are taken by those living with DMD.

The results of the survey will help Duchenne UK make informed decisions about the development of repurposed drugs as potential treatments for DMD and will facilitate clinical trial planning.

They survey is completely anonymous and will not ask you for any personal details.

The survey can be filled in via your mobile, PC or Laptop and will take no more than 20 minutes to complete.

You can complete the survey by clicking the link below:

https://www.surveymonkey.co.uk/r/YDXV37P

If you are a caregiver for more than one child/adult living with DMD please complete one for each child/adult as experiences for each may be different for each.

The survey will close at midnight on Tuesday 2nd January 2018.

Duchenne UK would like to thank you in advance for providing such valuable information and helping us move a step forward in increasing our knowledge in such an important area within Duchenne research.

For more information please contact research@duchenneuk.org

Complete the survey here

THANK YOU

December 23

Duchenne UK is a highly focussed, ambitious charity with a clear mission: to fund and accelerate research to find treatments and a cure for Duchenne muscular dystrophy. We have an incredibly lean team.

To run off minimal staff but create maximum impact, it is important we get input and advice from the right people.

We would like to dedicate today to thanking our incredible Scientific and Patient Advisory Board’s. Both are heavily involved in our funding review process. Providing us with their expert opinions on every project we fund.

Scientific Advisory Board

Our SAB is made up of some of the world’s experts in Duchenne muscular dystrophy. They bring with them a wide range of essential skills and knowledge bases, including scientific, clinical and drug discovery & development

We want to fund great science - projects that can improve our knowledge base, treatments that can reach the clinic and improve the lives of DMD boys in many ways - our SAB is a crucial part of this work.

Patient Advisory Board

Patient and parent involvement is at the heart of Duchenne UK. We believe that patients should be at the heart of drug development. Our Patient Advisory Board (PAB) is made up of people with DMD and parents of children and young adults of different ages, who advise and give feedback on the projects we are considering for funding.

NEW PODCAST EPISODE DECODING DUCHENNE

December 24

In this weeks episode of Duchenne UK's DECODING DUCHENNE podcast series, our host Clare Runacres interviews Lisa Kuhwald and Alex Johnson about the time of diagnosis. 

Alex Johnson is one of the co-founders of Duchenne UK. Earlier this year, Lisa joined Duchenne UK in a voluntary role as Patient Advocacy Officer. 

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To listen to the first four episodes follow the link to subscribe below or search "Decoding Duchenne" in your Podcasts app.

This is a really exciting new way to help raise awareness for Duchenne UK and Duchenne muscular dystrophy so please SUBSCRIBE on iTunes, rate the podcast and tell ALL your friends to listen too!

Decoding Duchenne is taking a break for the festive period. The next episode will be released in the New Year. 

SUBSCRIBE HERE

Thank you for following our 2018 Duchenneber campaign

December 25

Wishing you and your family a wonderful day.

Seasons Greetings,

The Duchenne UK team