Jon Hastie, CEO of DMD Pathfinders, shares what he has been doing with Duchenne UK to promote research and treatments for adults living with DMD:

DMD Pathfinders is proud to be working in partnership with Duchenne UK on our joint Adult Research Campaign, ensuring research and treatments can benefit everyone living with DMD, including adults like myself. Our partnership is based on a solid footing of shared values and recognition of each other's strengths.  Duchenne UK recognises the unique perspective of DMD Pathfinders which comes from being the only independent organisation run by adults with DMD for other adults.  Equally, DMD Pathfinders recognises the incredible value that Duchenne UK has brought to the DMD community, raising very significant funds and awareness, and investing them with great care to accelerate treatments for Duchenne.

DMD Pathfinders provides advice, information and advocacy for people living with DMD.  We advocate both for individuals in their lives as well as for the adult community as a whole.  That's why it's really important for us to push for treatments for DMD to be relevant and available to adults. 

When our campaign started at the beginning of the year, our priority was to understand the issue.  To do this, we mapped out over 40 organisations working on different treatments, all with a clear intention to eventually take these to market.  This has given us a far greater understanding of upcoming treatments and the potential they offered to adults. 

On the regulatory side, we spoke to regulators at the EMA to better understand their approach to regulating treatments for conditions such as DMD, and what steps could be taken at the regulatory stage to ensure a treatment can be accessed by adults when it comes to market.

The second stage of the project was to start engaging with many of the pharmaceutical companies themselves to understand their perspective on adults with DMD, to identify whether adults had been identified as a potential treatment group in their long-term plans.  We had some very successful conversations in this early stage, raising awareness of the adult community who are keen to see new treatments, and receiving some very positive feedback from the industry.

Our engagement over the last year has visibly pushed adults onto the agenda when it comes to research.  Duchenne UK has really helped to drive this forward with their announcement to invest in clinical trial capacity in an adult DMD clinic.  We were also pleased to collaborate with Action Duchenne on an adult symposium at their conference last month.

Right now, we have a far better sense of some of the barriers to adults accessing treatments, such as a conservative approach to trial endpoints.  It didn't take much research to identify that a six minute walk test will never provide the evidence needed to justify treating adults with DMD who cannot walk!  But what we also found is that endpoints such as respiratory function aren't as well recognised as they should be. 

While we continue to work on engaging with regulators and industry, having a better understanding of some of the barriers allows us to start to think about how we tackle them.  We need to campaign for change and raise awareness of the need for adults to access treatments - by creating a groundswell of patient activism to drive forward change.  For us, the next step has to come from adults with DMD themselves, to tell us what they want from DMD research, and what needs to happen to allow them to get involved. 

Personally, this campaign has shocked and excited me in equal measure.  Like many adults I've spoken to, I thought it was only a matter of time before adults would be able to access the benefits of research.  I was shocked to find out that some of the barriers we've identified could mean this never happens, unless people are willing to challenge the system.  I've also been excited by just how many treatments are being developed and the fantastic prospects for future generations.  It's given me even more determination to ensure this future can be realised for adults as well.

Jon Hastie, CEO, DMD Pathfinders