Navigating the ups and downs together

Navigating the ups and downs together

I am writing to you just following the news that NICE have published their decision not to recommend Vamorolone for Duchenne, a steroid alternative, for the treatment of Duchenne muscular dystrophy. Vamorolone, also known by the brand name Agamree, was approved by the Medicines and Healthcare products Regulatory Agency in January 2024 and I know that this news feels very disappointing for many of us. It highlights the long and uncertain road in securing better treatment options for everyone living with Duchenne and the need for our continued drive to be the voice of our community. 

John, our Science Communication Coordinator, has written an explanation of the decision and what it means at this point. Hopefully he has been able to answer a lot of your questions but please get in touch if you need more information.

Please feel free to reach out to us [info@actionduchenne.org] if you or your family are impacted by the news. We’re here to help and support you in any way we can.

Collaboration 

I have recently attended the PPI Conference in Italy, the Duchenne UK event in London and our Science Communications Officer has just returned from America where she attended the MDA Clinical and Scientific Conference. These events are an invaluable opportunity to work alongside the national and international organisations within the rare disease field. Action Duchenne are committed to being part of a strong multi-stakeholder approach, combining knowledge, experience and expertise to find a common strategy to connect with regulators and assist the evaluation and appraisal of potential treatments

Everyone living with Duchenne muscular dystrophy deserves a future with access to better treatments, and there is an urgent ongoing need for patient organisations to have their voices heard. We will continue to represent our community, to be their voice and to stand with them now and in the uncertain months ahead. I want to say to all of our families that we are here. We are fighting with you and for you, as well as offering you all the support we can along the way. 

Knowledge is power

The need for families to be supported has been echoed in the surge in demand for our services in the first quarter of this year. Our series of Winter Webinars, featuring a variety of Duchenne experts, offered information about how to talk to your children about Duchenne, physiotherapy, learning and behaviour in Duchenne and neurodiversity. We had over 100 families register for the webinars which are part of our ongoing commitment to provide families with the knowledge they need to make informed decisions. This continues with our Spring and Summer webinars, the first of which will take place on Saturday 13th April at 5pm with Annemieke Aartsma-Rus. She will explain how drug development processes work, with a focus on preclinical research. She will explain about the tools and the methods used and she will also illustrate how to use them in the right way and what can happen when the research is not done properly with examples from the Duchenne field. Finally, she will explain one therapeutic approach that is currently in the preclinical research phase: genome editing. I am very much looking forward to this session as part of empowering our community with the knowledge and understanding we need on this journey.

Fundraising Strategy

As part of our recent review of our strategy <Action Duchenne: Rebuild, Refocus and Reinvent>, our new Head of Fundraising has worked hard with the team on a new fundraising strategy and I am excited that this has now been reviewed and approved by our board of trustees. Going forward, our fundraising will be diverse, evidence based, mutually beneficial, integrated, sustainable and efficient. Our focus is on ensuring that we have a strategy that ensures the diversification of our income stream, the stabilisation of our finances going forward and that it cements our community engagement. Fundraising enables us to continue to expand our much-needed supporting families services and we recognise that our community and our supporters are the lifeblood of our charity. A key part of our fundraising strategy is ensuring that supporting us is a rewarding and positive experience

Come and be part of our amazing supporting families and fundraising community, we have many ways for you to be involved. We have new and ongoing volunteering opportunities for anyone who can share their skills and experience with us. You don’t need to have signed up for an organised event in order to raise money and awareness for Action Duchenne – many of our most successful events have been your ideas! Some of our 2023 highlights were a dinner-dance, a lemonade stand run by the children of one of our fundraising officers and 2 different music nights organised by a young man living with Duchenne. There are so many more examples to inspire you, and our dedicated fundraising team are always on hand to help you bring any of your ideas to life.

Our Team 

I was delighted to bring our whole team together on Tuesday 19th March for an in-person meeting. It is always wonderful to see the sharing of ideas, the support the team offers each other and the passion that drives our work. Our AGM was held on the same day, giving us an opportunity to share the impact of the work we have done with our valued members.

Becoming a member of Action Duchenne allows you to make a tangible difference to the lives of those living with Duchenne and their families, and also means that you become a part of the Action Duchenne family. You will be kept up to date on our projects and their impact, invited to ask questions at our AGM and vote on governance of the organisation and given advance notice and booking for our events, including our Annual International Conference.

Looking Ahead

We have recently launched our register of interest for the Annual Action Duchenne International Conference 2024, to be held on Friday 8th and Saturday 9th November at Leonardo’s Hotel in Hinckley. Our theme this year is ‘Education, connecting and wellbeing’ and we will have 3 simultaneous content streams addressing key areas on Duchenne muscular dystrophy, enabling you and your family to learn from the experts, build your support network and make time to focus on your mental and physical wellbeing.  

Registering your interest  will mean that you’ll be informed as soon as tickets become available. We will also keep you up to date with conference news, including the agenda and speaker confirmations. 

Close to my heart

I would like to share with you just some of the things that drive my continued determination to fight with the Duchenne community. Firstly, as we approach National Siblings Day on 10th April, I would like to acknowledge the many siblings in our community who are often the unsung heroes in our families. Many take on far more responsibility than their peers, understanding that the needs that come with Duchenne often overtake their own. While this invariably means that many siblings develop a level of empathy and understanding that they take with them throughout their lives, we know that it also comes with challenges. I want to take this opportunity to let all of our super-siblings know that they are seen and valued for all that they are and all that they do.

Please read the moving and insightful blogs written by two of our Duchenne siblings, Logan Kaye and Pilar Maestre

My final inspiration as I write this comes from the children and young people themselves, so many of whom are so close to my heart. Niall, who lives with Duchenne and whose family provide such valued support to our charity, won the Derry Credit Union Award for Courage this week. The courage and determination demonstrated by Niall and by all of those living with Duchenne are a constant reminder to all of us of what we are working towards. 

Our collective journey is never simple and there are many ups and downs, but we are with you through them all and our belief in our destination and in the power we have together remains unwavering.