Duchenne UK and DMD Pathfinders are conducting a joint campaign to ensure that treatments and therapies developed for Duchenne muscular dystrophy (DMD) are relevant and available to adults living with the condition as well as children.
The campaign is working with pharmaceutical companies, research funders and other stakeholders to push for adults to gain access to life-changing treatments.
As part of the campaign, DMD Pathfinders and Duchenne UK are:
- Identifying how existing pharmaceutical companies are engaging with adults with DMD in the research and development of new treatments
- Speaking directly with leaders in pharmaceutical companies urging for research and trials to include non-ambulant populations including adults with Duchenne
- Gathering evidence from adults with DMD to establish the need for treatments and assess the level of interest from adults in research
- Liaising directly with regulators and funders in the UK to raise awareness of the needs of adults with DMD
- Collaborating on a Duchenne information day specifically for adults to share best practice, new developments and valuable information
DMD Pathfinders is the first user-led organisation run by and for adults with DMD themselves, and provides the unique perspective of adults who have lived with the condition all their lives. As well as providing advice and information, DMD Pathfinders are determined to ensure adults are not left behind in the race for a cure.
Dr Jon Hastie, CEO of DMD Pathfinders, said:
“We are absolutely delighted to be working with Duchenne UK to ensure adults with DMD are firmly placed on the agenda for pharmaceutical companies, regulators and funders. This collaboration will bring parents and adults together to create strong, united voice to ensure that treatments for DMD are made available to all those living with the condition,
“For the first time we are now seeing treatments gaining regulatory approval and being rolled out to patients. Yet so far, the focus of research, development and clinical trials have been on preventing the progression of the disease in children while they are still ambulatory. Meanwhile, with life expectancy for adults with DMD continuing to grow, there is an increasing population of adult patients for whom data on safety or efficacy of treatments is sorely lacking.
“Depending on the speed of development of relevant treatments, the lack of evidence could mean that children currently living with DMD could end up unable to benefit from treatments when they become available. We cannot leave adults with DMD, many of whom have contributed extensively to fundraising and campaigning for research funds, out in the cold.
“There is an immediate urgency to ensure that pharmaceutical companies, regulators and funders understand the importance of gathering evidence and approving treatments that not only address the needs of children but also adults. This means considering adults at all stages of drug development including clinical trials which engage adults with the condition”.
Emily Crossley and Alex Johnson, Co-Founders of Duchenne UK said:
“Duchenne UK is equally delighted to be working with DMD Pathfinders on this joint mission to accelerate the availability of effective and safe treatments for all those living with Duchenne muscular dystrophy. Together we will ensure that the interests of adolescents and adults living with Duchenne receive the robust representation in the research community that they deserve’