Between 5th- 6th September, TREAT-NMD in collaboration with the UK Becker Muscular Dystrophy (BMD) Hub hosted an education and engagement day in Newcastle, UK for people living with BMD. This event was open to people living with BMD and their caregivers who are based in the UK, Ireland, and the Netherlands. The day included expert-led sessions on care, genetics & research, interactive workshops and support discussions and an opportunity for attendees to connect with others who understand their journey.
Newcastle University and Duchenne UK are developing patient-centred resources in PaLaDIn to facilitate fair and equitable access to trials and speed up the development of effective treatments and care for neuromuscular diseases. The resources include:
- Clinical Trial Finder (CTF) to identify research opportunities
- Central Recruitment Database (CRD) to enable recruitment
These two resources will inform the development of the Interactium® data platform that is being developed as part of the PaLaDIn project.
Partners from Newcastle University attended the BMD education and engagement day to hear from those in the Becker Muscular Dystrophy community, to ensure that these resources meet their needs. Part of the event included a session involving a poll of attendees to ask them a range of questions about BMD and their opinion on priority areas.
The session started by asking the attendees what they thought were the main priorities for the BMD patient community. 16 participants fed back that information, community, medicine and treatment were the main priorities, as displayed in the word cloud below.
Participants also fed back that clinical trials and research opportunities are gaps in the clinical care provision for BMD. Three quarters of participants polled said that they find it difficult to locate information about the BMD Condition when they try to search for it and that clinical trials and research opportunities were gaps relating to information currently available.
When asked to rank issues the BMD community face in order of importance, participants stated that not enough clinical trials, not finding out about trials and difficulty in participating in trials were the most pressing.
Participants were presented with information about two tools that have previously been developed in DMD to help the community locate and access information on research. The DMD Hub Clinical Trial Finder and the Central Recruitment Database. When asked if similar resources were developed for BMD, participants were overwhelmingly in support of this, with 94% in favour.
The insights gathered from this engagement day will directly inform the development of BMD-specific resources within the PaLaDIn project. With strong community support for tools like the Clinical Trial Finder and Central Recruitment Database, Newcastle University and partners are committed to creating accessible, patient-centred platforms that address the information gaps and trial access barriers identified by the BMD community. By working collaboratively with patients, caregivers, and clinicians, we aim to ensure these resources truly meet the needs of those living with BMD and accelerate progress toward better treatments and care.
