Reflections and Hope

As we approach the festive season and the end of 2024, I have been reflecting on a year filled with triumphs and challenges. It has been a time of uncertainty, but also one of progress, resilience, and hope for the Duchenne community. In this blog, I would like to share some key highlights from the year and our aspirations for 2025. 

Support families across the UK – All-Though Support Continues

Throughout 2024, we remained committed to supporting families from diagnosis and beyond. Thanks to new grant funding from trustees, foundations, and pharmaceutical partners, we have expanded our support to reach more families across the UK. This funding will help us ensure that no family feels along on their Duchenne journey. Read more about our funding news here.

We have been very aware of the additional challenges to many of our families of the rise of the cost of living this year, and we’ve worked hard to adapt the format of our delivery. 

This year, we reduced the number of in-person events to ease the pressure on families. Instead, we offered alternative ways for families to stay connected. Our regular webinars have covered a large range of topics. Delivered by Duchenne experts in areas such as psychology, neurodiversity, physiotherapy, drug research and development and more, they have given our community the opportunity to increase their understanding and feel empowered to make informed decisions. This is particularly important to me as there is increasing uncertainty facing Duchenne families around upcoming treatment options. In the last few months we have seen the announcement of the Early Access Programme for Givinostat, and NICE’s approval of Vamorolone as a new generation of steroid. As a charity we have continued to fight tirelessly for increased access to treatment options for everyone living with Duchenne and both announcements represent groundbreaking steps forward. However, there is still a long way to go in fighting for equitable access for everyone, and I want to reassure you that we are unwavering on our commitment to advocating for this. For the families we support,  these announcements often bring with them yet more unanswered questions, concerns and further complexity around the decisions that parents need to make for their children. It is vital that we continue to provide opportunities throughout our science education programme for our community to be able to ask questions, gain understanding and feel confidence in their own knowledge. 

Listening to our community

Our counselling programme has been a highlight of 2024 and I am immensely proud that we have been able to offer Duchenne parents and carers 8 weeks of online counselling with a professional counsellor. The group format of this allows for strong peer to peer connections to be formed and for support networks to continue to grow long after the programme has finished. Our Pit Stop area at our 2024 International Conference in November, an area dedicated to the wellbeing of those attending, really highlighted our ongoing commitment to providing emotional and psychological support for families wherever they are on their Duchenne journeys. By listening to both what families really need and to the practical challenges that this year has brought, we have been able to adapt what we provide and we will take this forward into the new year. 

In talking to our community, we know that having the right support and understanding for children and young people living with Duchenne in school can be challenging. In response to this, while our in-person science education events have been reduced this year, we have expanded our work with schools. As part of our vision as a charity to create a more inclusive society, Action Duchenne has provided whole school assemblies, interactive classroom workshops and staff training as well as support with the EHCP process to 6 schools this year, and there are plans to increase this into 2025 and beyond. The day to day challenges facing our families are always at the forefront of our work, and we hope that our support for schools can ensure that young people living with Duchenne can meet their full potential and that this will in turn reduce some of the ongoing stress for parents and carers. 

“The session was really informative and helped myself and the staff tackle questions we all had but didn’t know how to ask. We feel more informed and prepared to deal with Duchenne.” School Staff Member

Supporting young people living with Duchenne is a key part of our work and I was delighted that our Creche and Hang Out areas at our conference this year were the busiest they have ever been. There were two full days of activities for children and young people living with Duchenne and their siblings who had the chance to come together, have fun and develop lasting relationships. Connection with others through shared experiences has always been a highlight of our conference for families, and it is particularly special to witness this taking place for our children and young people. 

Community Fundraising

While the challenges of the cost of living 2024 for all of us have been seen across  our community fundraising as it has for many in the charity sector, we are incredibly grateful for the ongoing commitment and dedication of our community. As well as those taking on challenge events for us, a particular highlight this year was the wonderful (albeit wet!) day at the Parallel Windsor Festival of Inclusivity. Meeting some of our amazing families and seeing them able to participate in the variety of events on offer while connecting with each other made it a special event for me. Our 79 Challenge for World Duchenne Awareness Day ran for its second year in 2024, and our participants ranged from individuals, families, schools and businesses. 2024 was the first year that 7th September was officially designated by the UN as Duchenne Awareness Day, and this gives me renewed hope for increased  global awareness and understanding, driving forward standards of care and treatment options. 

I was deeply honoured to end the year with a real highlight, visiting JLR Solihull on 13th December to receive a generous donation from the company. Action Duchenne was chosen as one of the charities for JLR’s Season of Soul initiative and Ian Taylor, Duchenne dad and JLR employee, shared with the JLR team the challenges faced by families living with Duchenne as well as the impact of the work we do. Read more here  This demonstrates the incredible impact our community can have in raising awareness of Duchenne and the vital work we do. 

This year has been difficult for many and the charity sector has not been exempt from these challenges. However, your unwavering support has allowed us to continue our vital work.  As a small charity, we rely heavily on our community fundraising for us to be able to continue to provide much-needed support to our families and anything you can do, however big or small, is always welcome and makes a tangible difference. Thank you for standing with us. 

If you would like to get involved and help us make a difference, please consider supporting us. Visit our website to learn more about how you can contribute. 

Looking ahead to 2025

As we look to the new year, we are driven by our vision of an inclusive, supportive, and empowered community. Together, we will continue to advocate for equitable access to treatment, support families through the challenges they face, and provide opportunities for connection and education. 

Thank you for being part of our journey this year. Your strength, courage and dedication inspire everything we do. Wishing you and your families a happy festive season and a bright and hopeful 2025.