Five Years at Action Duchenne
This month marks five years since I joined the team at Action Duchenne, and it has been an incredible journey. From both an organisational and community perspective, these years have been filled with challenges, achievements, new experiences, and continuous learning, all driven by our mission. While much has changed, our unwavering commitment to supporting the Duchenne community and striving for a future where lives are no longer limited by Duchenne remains as strong as ever.
Overcoming challenges
One of the biggest challenges early in my time at Action Duchenne was the pandemic. Families affected by Duchenne had to shield, making access to vital care and support even more difficult. We acted quickly, moving our services online so families could still access resources, webinars, and our Annual International Conference. It was a tough time, but the dedication of our team and the resilience of our community ensured we could keep providing the support that mattered most.
“I am proud to have led the team this year, through the complexities of remote working, home schooling, the decimation of our traditional income streams, the exponential demand on our services, new funding, new projects, and a completely new landscape for the economy. “ From my blog in February 2021
Action Duchenne Annual International Conference
Despite the unexpected challenge of delivering the first two Annual International Conferences virtually, this event has been a highlight of each year during my time with Action Duchenne. Whether virtual or in-person, these events bring together those living with Duchenne, families, clinicians, researchers, pharmaceutical companies and equipment providers to share knowledge, experience and build support networks. It has been a privilege to witness this and to be a part of providing this opportunity over the last five years. Delivering Welcome Remarks to open this year’s conference alongside Ravi Mehta, who works for Action Duchenne and also lives with Duchenne, was a personal highlight for me.
Expanding our ‘All-through Support’ – supporting families at every stage of life
A milestone in my journey with Action Duchenne came with the funding from the National Lottery in 2021 which enabled us to develop put our ‘All-through Support programme. We listened to the unmet needs within the Duchenne community, and designed a programme to fill the gaps in support for our families. Based on providing ongoing support from the point of diagnosis right through to end of life, bereavement and beyond, this programme encompasses many aspects of emotional support, including targeted support for newly diagnosed families, 1-1 and peer to peer support as well as regular online support groups, science education to enable families to make informed decisions and support for young people living with Duchenne.
This programme underpins our continued work, and supporting families through all of the challenges they face throughout their Duchenne journey is fundamental to our charity. Working with partners such as The Pathfinders Neuromuscular Alliance, Treloar School and College, and MDUK has helped us extend this vital support to even more families.
Helping young people gain independence – ‘Yes I Can’
The Transition to Adulthood project, led by Ravi Mehta, has helped many young people living with Duchenne develop essential skills for independent living, higher education, and employment. One of the most rewarding aspects has been the residential weekends, where young people challenge themselves through activities, gain confidence, and build friendships with others who understand their experiences.
“The young people took part in a variety of adventurous activities, challenging themselves and pushing themselves out of their comfort zones alongside others who really understand. I was inspired by witnessing their achievements, joining their fun and seeing the confidence they gained. I came away from this weekend more convinced than ever of the potential our young people have and more determined that our work must continue to support them to achieve it.“ From my blog after the residential weekend in 2023
Advocacy for better access to treatment
Advocacy continues to be at the forefront of our work, and we are proud to have been alongside other Duchenne charities such as Duchenne UK and MDUK to be part of fighting to make new and emerging treatments available to those who need them most. While we have had recent success with the NICE approval of Vamorolone and the Early Access programme for Givinostat, we know that there is a long way to go and that there are many questions to be answered. 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. 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.
Fundraising and Community Support
The strength of our community is evident in their fundraising efforts. Despite the challenges of the pandemic and the rising cost of living, people have continued to raise vital funds through events such as online fundraisers, marathons, and local initiatives. Running the Vitality 10km in 2023 alongside our supporters was a special moment, demonstrating the dedication and generosity of those who help us continue our work.
Looking ahead to 2025, we are excited about upcoming events, including the second Charity Dinner Dance on 22nd March and the Parallel Windsor Festival of Inclusivity. These events not only raise funds but also bring people together to celebrate resilience, hope, and progress.
We would love to have your support to help us to continue our work. Community Fundraising is vital to the continuation of our much-needed work, and there are many ways you could help. Find out more about signing up for one of our events, creating your own fundraiser, making us the charity of the year for your school or business or becoming a valued member of our charity on our website.
Acknowledging our trustees
Our trustees play a crucial role in guiding and supporting the charity. We are grateful for the contributions of all our trustees, particularly Gary Fegan and Mark Silverman, who recently stepped down after many years of service. “We wanted to sincerely thank you for the years of work you have given to the charity, and the unstinting energy and enthusiasm you have always given to improve the lives of people living with Duchenne and their families. Your input has made a real difference to many people’s lives and that is a great achievement. We hope we will have opportunities to work together again and wish you all the very best for the future.” Vicky, on behalf of the Board of Trustees.
We also warmly welcome Marion Main to the Board. With her vast experience in neuromuscular physiotherapy and clinical research, she will be a valuable addition to our team. Many of you will have been to Marion’s sessions at our Annual Conferences where she has been a regular speaker. She is a highly experienced neuromuscular physiotherapist previously with the DubowitzNeuromuscular Centre at Great Ormond Street Hospital (GOSH) in London, having retired from the NHS in 2024. She currently has an associate contract with UCL to continue teaching and advising on clinical research. We are really excited about the expertise and experience Marion will bring to the charity and we look forward to working with her.
“On behalf of the Board of Trustees at Action Duchenne, I am delighted to warmly welcome Marion as a new trustee. Her wealth of experience and expertise and her dedication to our mission will be invaluable to the charity and to the families we support as we continue to work towards a world where lives are no longer limited by Duchenne muscular dystrophy”. Vicky, on behalf of the Board of Trustees.
Looking forwards
We continue to develop new initiatives to support families. Our monthly online support groups and counselling programmes for parents and caregivers are an important part of this work. Online support groups for Dads, Mums and Grandparents take place during the first week of every month, and are an invaluable way to connect with people who truly understand and to increase your support network. We are starting a new group counselling programme following the success of the previous ones. This programme is holding space for the emotional burnout that comes with the increased care needs of your young people. We know the juggle of supporting your sons with accessing the world in education, independence, careers and socially has big implications on your capacity mentally and physically. It’s important that you have time for yourselves too, we want to offer an 8 session programme that brings you together with a group of parents/carers in a similar stage of the DMD journey. Please get in touch with kellymolkenthin@actionduchenne.org for more information.
We are also excited to expand our All-Through Support Programme into Scotland, thanks to new funding from the National Lottery Community Fund Scotland. Find out more about this and how you can get involved.
We have recently launched our 2025 Webinar Series, bringing national and international experts directly to families. Covering topics from neurodiversity in Duchenne to bone health, steroids, and the drug approval process, these sessions run from March to October. The first webinars, delivered by renowned developmental paediatrician Professor David Schonfeld, will take place on 4th and 18th March. Topics include The Impact of Diagnosis and Coping with Grief and Loss, aimed at those navigating a new diagnosis or adjusting to a new stage of the condition.
The team here is looking forward to joining with the global Rare Disease Community on 28th February, and invite you to join with us in raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and carers. The theme this year is “More than you can imagine; an anthology of rare experiences” and if you would like to share your experiences of living with a rare disease as a parent/carer or as someone living with Duchenne, we would love to hear from you. Find out more here.
Moving Forward Together
Reflecting on the past five years, I feel incredibly grateful for the ongoing support of our team, community, partners, sponsors, and fundraisers. Your dedication drives our progress and strengthens the impact of our work. Most importantly, to the Duchenne community – thank you for inspiring us every day. Your resilience and determination motivate us to keep pushing forward.
Together, we move ahead with purpose, determination, and hope for a future where Duchenne no longer limits lives.
