Fighting for the future
While we continue our much needed day to day work supporting our families, I want to share with you the recent developments behind the scenes. I am passionate about being part of the fight for access to new treatments for Duchenne and at Action Duchenne we are driven to advocate for access to the best possible available treatment to the Duchenne community. With the recent development of promising technologies in the sector of rare paediatric conditions, there is a need to campaign for the access to treatment and be the voice of the community. Our team has been championing engagement with multiple stakeholders and regulatory bodies to drive conversations to improve access to therapies for people living with Duchenne muscular dystrophy.
On the 15th of March, I was incredibly proud to be able to represent the Duchenne community at the Office for Market Access (OMA) Collaboration Space Pilot meeting. OMA is an early engagement that takes place between patient organisations, clinical experts, industrial partners, and other stakeholders to discuss the current challenges faced by upcoming technologies. This is to ensure preparedness and sustainability of the technologies on the horizon. Amongst other factors, the main points of discussion were the care pathway, commissioning, service delivery and HTA considerations for technology development. Action Duchenne advocated for an approach to therapies that would apply to all, regardless of their age, access to a standardised care pathway regardless of where you are based in the country.
On the 26th of April, I was honoured to be invited to attend a parliamentary meeting to explore potential measures to improve the access environment for advanced therapies (ATMPs). Multiple stakeholders were present at this prestigious platform including senior representatives from National Institute for Health and Care Excellence (NICE), NHS England, Genetic Alliance UK, Cell and Gene Therapy Catapult, Department of Health and Social Care, and the BioIndustry Association. This was a great opportunity to discuss, brainstorm, collaborate and have transparent dialogue around the ways to improve access to ATMPs, and share insights to ensure a sustainable and balanced approach for the future. The current challenges faced by the access environment for ATMPs were discussed with great engagement around policy development and solutions to overcome any existing gaps.
Action Duchenne is proud to be a part of these effective and insightful discussions to pave the way for a better future for the Duchenne community. As a community led charity, we strive to share the views of the families living with Duchenne to the multiple stakeholders in the field.
Science-on-Tour 2023 and Friday Hive: Supporting families
We ensure that our work enables us to engage with our community face to face, meaning that we ensure that we know the real challenges they face. The Science on Tour programme continues to deliver small group workshops around the country, giving us the opportunity and privilege of meeting with many of our families alongside those supporting them.
Support sessions like the virtual Friday Hive provide a great platform to engage with the community and hear about the challenges being faced on the ground and receive support.
Action Duchenne International Conference 2023, 10th and 11th of November
I am very excited that the team is getting ready to launch the registration for our 21st Annual International Conference, to be held on the 10th and 11th November 2023 at Leonardo Hotel, Hinckley Island, Leicestershire. The conference is an unmissable opportunity to bring families, clinicians and pharmaceutical companies together and I hope you’ll join us for what is a highlight of the year for Action Duchenne.
International Conference and Community – Action Duchenne
Our dedicated team is putting together the conference registration site to be launched very soon. We plan to run a pharmaceutical company question and answer session. Due to the current UK regulations and ABPI code of conduct, no questions can be asked on the day. Therefore, Action Duchenne would like to request the Duchenne community to share any questions they have for pharmaceutical companies with us beforehand. Please be mindful that due to the current UK regulations no drug/product or clinical study specific questions can be answered by the companies.
Please send your questions for the pharmaceutical companies’ question and answer session, taking place at Action Duchenne’s International Conference 2023 to:
mehreen@actionduchenne.org and sofiya@actionduchenne.org
We are clear in our mission to strive to improve the quality of life of people living with Duchenne and always being the loud and strong voice on behalf of the Duchenne community.
Vitality London 10,000 Supporters event, 24th September 2023
If I haven’t mentioned it to you already, I am training for the 24th September 2024 Vitality London 10KM. I am not a runner, and a 10,000 run has not been as straightforward as I had thought but I am determined to make it to the start line and finish line in September. I have the privilege of being the National Director of Action Duchenne, and I am incredibly proud to be raising money for Action Duchenne, not just because the Charity has injected over £10 million into Duchenne research, but because of the Charity’s focus on supporting families to live with the condition. Their support is absolutely vital to the entire community. Up and down the country there are several thousand Duchenne families – not just parents and siblings, but also grandparents, aunts, uncles, and family friends – that have been helped immeasurably by the team here at Action Duchenne.
The charity has 40 places in the event and we are looking for people to join our team, and hopefully have a picnic in the afternoon. If you’d like to join me and other Action Duchenne Supporters on Sunday 24th September 2023, please sign up at the link here: Vitality London 10,000 – Action Duchenne Your 10000race efforts will be helping support us to get one step closer to beating Duchenne!
The Vitality London 10000 is a brilliant event with a great atmosphere and I would love to have a full team of Action Duchenne runners taking on the challenge with me, coming together to raise money and awareness. Last year’s runners absolutely loved it, as did our cheer squad. It will be a fantastic event with its iconic route, entertainment all the way round and finishing outside Buckingham Palace.
Hear from our mums
Our Project Engagement and Marketing Assistant Lizzie recently shared her experiences of being a Duchenne mum to 9 year old Sebastian as part of our new ‘Turning Point’ project. This project will support young people living with Duchenne as they go through the transition from primary to secondary school, which often coincides with a change in mobility. Read her blog here and if you’d like to share your experiences, please email dawn@actionduchenne.org. We’d love to hear from you.
Charity Dinner Dance
I want to finish this month with a focus on the amazing ability within our community to turn devastation into positivity and to channel sadness into strength. Ruth and Ian Taylor held a charity dinner dance to raise money for Action Duchenne in March 2023, held at Aston Villa Football ground, attended by over 300 people and comprising a 3 course meal, raffle, auction with live bands, a DJ and a lot of fun. I was honoured to attend alongside 3 other members of the Action Duchenne team. A full article about the event can be found here, along with a video interview with Ruth and Ian who are truly inspiring: “Making something positive out of something negative” – Action Duchenne.
Supporting YOU
It is for families like them that we continue our fight, to get their voices heard and to create real change for their future.
We are here to support you and your family. Please do Contact us – Action Duchenne, and we will get in touch with you.
Until next time, please take care and best wishes,
Florence
