WELCOME TO THE
WELCOME We look forward to welcoming you to our 20th International Conference. While we regret that the COVID pandemic has once again prevented us from holding the conference in person at Hinckley Island, we are excited about the opportunities of holding an innovative virtual conference. This is the second time the conference will be organised with our expert colleagues from the UK. Holding the conference virtually will be a great opportunity for Action Duchenne to update on research and trials, standards of care and living with Duchenne as well as using digital and social media during the pandemic to reach a wide audience across the world. This past year, we have all become more accustomed to living our lives online, attending virtual events, speaking with loved ones on Zoom and even conducting clinical appointments virtually. It has become the ‘new normal’ and although we are pleased to offer the Duchenne community the chance to come together virtually, we very much look forward to the day we can hold our conference in person again. Throughout the conference, you will hear from people living with Duchenne and Duchenne families, who will share their personal experience and knowledge. We hope you will come away from the conference with knowledge and power; further equipped with information to help you make important decisions. We hope you will have your questions answered, giving you additional confidence to take on the next year of your Duchenne journey. We are using brand new technology to create a more user-friendly and immersive experience for you. You will have the chance to take part in competitions, meet new people in the social area and, of course, watch and interact with expert speakers, pharmaceutical companies, exhibitors and clinicians. With the support of our valued sponsors; (Elite) PTC, Sarepta, (Gold) Pfizer, (Silver) EdgeWise, (Bronze) Solid, Dyne and Capricor, we are once again, able to offer free conference tickets to all UK and international Duchenne families. In addition, we are delighted to welcome back SMART Wheelchairs, Centrobed and Queen Alexandra College to our exhibition area. Thank you to all our speakers for taking part this year and a special thank you to Dr Jon Hastie of The Neuromuscular Pathfinder Alliance and our partner organisations, Muscular Dystrophy UK and The Paskin Children’s Trust for their valuable contribution to the conference content. The entire Action Duchenne team - our staff, trustees and dedicated volunteers - are here to help you get the most out of your time at the conference. You can get in touch with any of us at any time using the chat facility on the platform. As always, we are here to support you. I look forward to welcoming you to our conference!
Florence Boulton Action Duchenne National Director
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IMPROVING LIVES SEARCHING FOR A
CURE
ABOUT ACTION DUCHENNE
OUR VISION
We are a community-led charity, putting our Duchenne young people, adults and their families at the forefront to meet their needs.
Our vision is clear: a world where lives are no longer limited by Duchenne muscular dystrophy. We have three core objectives and are proud that 87p in every £1 raised is spent on our charitable activities:
Our expertise is driven by the lived experience of our team: half of our staff and trustees have a direct connection to Duchenne. We combine that experience with expertise from across the business, research and charity sectors to give us an unparalleled ability to deliver complex projects efficiently and successfully.
- Funding research for everyone living with Duchenne - Cutting-edge science education programmes - World-class support for Duchenne families
Photo: David Mace
CONTENTS
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WELCOME 1 ABOUT ACTION DUCHENNE 2 OUR VISION 2 WHAT WE HAVE ACHIEVED 3 HOW TO USE THE CONFERENCE PLATFORM 4 LIVE CONFERENCE AGENDA 5 THANK YOU TO OUR SPONSORS 6 VIDEO LIBRARY 7 MEET EXPERT SENCO 7 HOW YOU CAN HELP 8
WHAT WE HAVE ACHIEVED
50+ 50+ 22+
5 1,000+ 40,000
Duchenne Science LIVE episodes
nations represented
of online viewers of our science education channel
expert speakers from around the world Duchenne patient organisations represented
unique pageviews of our COVID-19 support webpage
What’s next?
What’s next?
- Regional science workshops across the UK - Online support and science education
- A dedicated programme for young people, adults and their families living with Duchenne - Online science shows
2000+ 6 30+
30+ 37
young people, adults and their families given direct, bespoke, support
support webinars
blogs published sharing
families’ lived experience
What’s next?
peer-to-peer support groups
- Dedicated project supporting parents through diagnosis - Aspirational support project for young people transitioning into adulthood - Complementary end-of-life support project - Regional peer-to-peer support groups - Supporting Duchenne families
community events nationwide provide local support
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HOW TO USE THE CONFERENCE PLATFORM TO ACCESS THE CONFERENCE PLATFORM: Visit https://adregistration.org.uk/conference and register
WHAT THE CONFERENCE PLATFORM OFFERS: Live sessions - there is one stream of live sessions running between 10:00 and 21:00 GMT on Saturday 13 and 10:00 and 18:00 on Sunday 14 November
A chance to ask questions - during live sessions, you can ask questions via the chat box to the right of the video stream, or Zoom in live to join the speakers face to face using the Call Now button underneath the video screen.
Recorded content - access more detailed information at your own pace within the video library area.
The Meeting Place - want to chat to other attendees? The Meeting Place virtual social area is open throughout the conference. Drop in during the breaks to speak with others.
Gamification - get points and build the 79 exons on your virtual Dystrophin gene through taking part in different activities. More points = prizes!
Polling - please take the time to give your feedback when prompted, so that we can continue to improve our events for you.
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LIVE AGENDA
SATURDAY 13 + SUNDAY 14 NOVEMBER
SATURDAY 13TH NOVEMBER. ALL TIMES IN GMT TIME
SUBJECT
SUMMARY
10.00 - 12.00 Treating Duchenne and Standards of Care
The Duchenne Standards of Care is a document that sets out the care that people living with the condition should receive. You will hear from leading clinicians from around the world about a range of topics from bone health, heart drugs and use of steroids.
12.00 - 13.00 Break / social time
We know how much you gain from interaction with families, clinicians and researchers, so please head to The Meeting Place during the breaks and social time. There will be lots of opportunities to gain points in our conference game, update your details on the UK DMD registry, meet the speakers, visit the exhibitors, speak with conference partners and share support and knowledge with each other.
13.00 - 15.00 Latest clinical trial news
Professors Francesco Muntoni (UCL) and Volker Straub (Newcastle) have been the lead investigators in many of the Duchenne clinical trials within the United Kingdom and globally. They will host a session exploring the latest advances and results from clinical trials around the world, where you will have the opportunity to pose your questions to the expert panel.
15.00 - 16.00 Break / social time
Remember to head over to The Meeting Place to meet the exhibitors, speak to the experts and chat with Duchenne families.
16.00 - 18.00 Caring for older children and adults living with Duchenne
It is important that adults receive the care and services they need to stay as healthy, independent, and active as they want to be. Hear from leading experts covering topics such as; respiratory management, the benefits and challenges of tracheostomy, gastrointestinal issues and management, nutrition and diet, glucose and exercise, the benefits and challenges of PEG tubes.
19.00 - 21.00 Evening social: Living as an adult with Duchenne
After a quick break, continue the conversations raised earlier in the ‘Adults standards of care’ session. The evening social is a private session for adults living with Duchenne and their friends/siblings to chat and meet other like minded people. Expect frank and open conversations around topics such as relationships and dating, gaming, finding a job and living independently.
19.30 - 21.00 Music and me
Join the stars of our “Music and Me” podcast for an evening of music and chat. View the agenda in the conference platform and you can add sessions to your customised agenda by clicking the ticket icon 5
SUNDAY 14TH NOVEMBER. ALL TIMES IN GMT TIME
SUBJECT
SUMMARY
10.00 - 12.00 The effects of COVID-19 on Duchenne Research and Care
The pandemic has seen huge changes in how medical care is delivered, and how drugs are developed, tested and approved. Our expert panel will be discussing the challenges and successes of the move to hybrid trials with appointments at home, virtual clinic appointments, faster regulation and increased drug manufacturing capabilities.
12.00 - 13.00 Break / social time
Remember to head over to The Meeting Place to meet the exhibitors, speak to the experts and chat with Duchenne families.
13.00 - 15.00 Dystrophin and the brain, learning and behaviour
Starting with the science, you will find out why dystrophin is in the brain, what is its purpose and what happens when it is missing. Explore learning and behavioural issues in Duchenne with our panel which includes a leading SENco and behavioural experts. Find out about how to navigate school and how to manage and support individuals and families living with the condition.
15.00 - 16.00 Break / social time
This is your last chance to clock up points in the conference game! Visit the exhibitors, update your details on the DMD Registry and share your experience with others. Great prizes are up for grabs, so make the most of the final hour’s break!
16.00 - 18.00 Living with Duchenne
Hear from young people and adults living with Duchenne as they share their personal experience and knowledge. Gain practical information from our partner organisations on topics to help you navigate your journey such as housing adaptations and benefits. Get inspired and find out what is possible to help you meet your aspirations.
Thank you to our sponsors:
View the video library in the conference platform where you can view content by topic, audience and speaker!
VIDEO LIBRARY The video library on the conference platform gives you access to a vast library of content that we’ve recorded in the run-up to the main event. You’ll find videos on the content of each of our live sessions and more.
All video content will remain available after the conference.
MEET EXPERT SENCO Clair Warner will be available to meet you between 15:00 and 18:00 GMT after her session. Talk to her about your young person’s learning and behaviour, discuss the process for your Education and Health Care Plan, get practical advice on navigating school and how to manage and support your young person. View Clair’s step-by-step EHCP guide in the video library
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Photo courtesy of Lowestoft Journal
Supported by friends and family, Molly, Zak and their little boy Drew (2) took action by organising their First Steps 7 miles walk
HOW YOU CAN HELP The Action Duchenne International Conference is a truly unique opportunity to engage with the Duchenne community. Here are the ways you can get involved in next year’s event: Become a sponsor - we are only able to provide world-class sessions and expert speakers free of charge to the global Duchenne community through the generous support of the conference sponsors. Put us in touch with potential corporate sponsors and we’ll do the rest! Exhibit at the conference - we make it easy and cost-effective for you to showcase your products to hundreds of Duchenne families from across the world. Volunteer - join our incredible conference volunteer team, who provide invaluable support to our staff for this event. Offer pro bono support or services - you can help in a number of ways, from proofreading to printing our conference materials, photography to video production. We are keen to hear from experts who would like to help us deliver the conference in 2022. Please contact us on info@actionduchenne.org to discuss your involvement in the conference and to make plans for 2022! Donate now 8
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John Miller, dedicated Grandad and our Scottish Advocate, created a fundraiser for us to celebrate his 82nd birthday
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ACTION DUCHENNE LIMITED 49-51 East Road, London N1 6AH • 020 7250 8240 • info@actionduchenne.org • www.actionduchenne.org Registered Charity No: 1101971, Scotland: SC043852
