Pioneering data platform launched to accelerate research and improve access to care and treatment

We have launched Duchenne UK Connect, a new digital platform and app designed to support people living with Duchenne muscular dystrophy and their families while accelerating research into the rare genetic condition.

Our secure platform combines practical tools, trusted care information, clinical trial opportunities and a patient registry in one place, empowering families to manage Duchenne while contributing valuable real-world data to help improve care and advance treatment development.

We have funded and developed Duchenne UK Connect in collaboration with the Duchenne community, clinicians and researchers at the John Walton Muscular Dystrophy Research Centre at Newcastle University. It brings together a patient registry, care, clinical trial and research opportunities, and emergency health information within a single, user-friendly platform.

Addressing the needs of the Duchenne community‍

As a charity focused on driving advances and progress for people living with Duchenne muscular dystrophy in the UK, we have designed Duchenne UK Connect to help families access trusted information, best-practice care guidance, and opportunities to participate in research and clinical trials, regardless of where they live.

We will run Duchenne UK Connect in collaboration with Newcastle University. The platform incorporates the work of Duchenne Care UK and Duchenne Hub UK, our joint programmes with the John Walton Muscular Dystrophy Research Centre.

Emily Reuben OBE, Chief Executive and Co-founder of Duchenne UK, said:

"Data has a vital role to play in advancing research and improving outcomes for people living with Duchenne. Duchenne UK Connect gives families practical tools they can use today, while helping generate the insights researchers need to develop new treatments and care in the future. By bringing together trusted information, research opportunities and emergency health information in one place, we hope to make life easier today while helping transform outcomes for future generations.

“Moreover, as a mother to a son with Duchenne, I know first-hand the value of being able to access key health information and clinical guidance in an emergency, and I truly believe that this platform has the potential to save lives.

"Importantly, Duchenne UK Connect puts people affected by Duchenne at the centre. Patients and families remain in control of their data. They choose their level of participation and how their data is used. We recognise everything patients and families affected by Duchenne go through and the bravery it takes to share personal health information and detail experiences. We are therefore committed to ensuring that whatever data people share is put to good use and benefits our community.”

Bringing care, research and emergency information together‍

Duchenne UK Connect consists of four modules: Care, Registry, Research and In Case of Emergency, with users choosing which modules they wish to participate in.

Through the platform, users can:

- Store and manage important health and care information securely.

- Create and share an up-to-date emergency health profile when urgent medical information is needed.

- Access trusted care recommendations and family resources developed through Duchenne Care UK.

- Find clinical trials and research studies across the UK and register interest through Duchenne Hub UK's Central Recruitment Database.

- Complete questionnaires sharing data and insights, with consent, to support research, improve services and accelerate treatment development.

The platform is available online and through dedicated apps on the Apple App Store and Google Play Store, giving families access to information and support whenever and wherever they need it.

Alex Johnson OBE, Founder of Duchenne UK and Chief Executive of Joining Jack, said:

"Over the past 14 years, Duchenne UK has helped transform the landscape for people living with Duchenne, from improving access to clinical trials and developing standards of care to supporting the approval of innovative treatments.

"Duchenne UK Connect is the next step in that journey. It brings together the resources, services and tools we have built into one place while developing a powerful new source of real world, patient reported data that will help drive future progress."

Putting patients in control of their data‍

Participation in Duchenne UK Connect is entirely voluntary. Users decide which modules they join, what information they share and how their data can be used. We have developed the platform with robust governance, strong data security and user-controlled consent at its core.

Our new platform also marks a major step forward for Duchenne research in the UK, replacing and expanding existing Duchenne registries while integrating Duchenne Hub UK's Central Recruitment Database into a single national resource.

As the platform grows, approved researchers from universities, the NHS, charities and industry will be able to apply for access to anonymised datasets through a rigorous governance process designed to ensure all research benefits the Duchenne community.

Professor Michela Guglieri, Principal Investigator for Duchenne UK Connect, Professor of neuromuscular disorders at the John Walton Muscular Dystrophy Research Centre at Newcastle University, and honorary Consultant at Newcastle Hospitals NHS Foundation Trust, said:

"Duchenne UK Connect represents a unique resource for patients and families to access information on care and clinical trials, express their interest in participating in research, and access key clinical information and recommendations in case of emergency. By bringing these resources together in one platform, it will help support more equitable access to care and clinical research for everyone living with Duchenne in the UK.

"Importantly, it also offers patients and families the opportunity to actively contribute to improving our understanding of Duchenne, sharing their experiences and priorities to help address important unanswered questions and ultimately improve care and outcomes. I am very pleased to be part of this initiative."‍

The launch comes as healthcare organisations around the world increasingly invest in digital technologies that enable patients to play a more active role in managing their health while generating vital evidence to improve researchers' understanding of rare diseases, advance research, bring forward new treatments and improve outcomes.

You can learn more about Duchenne UK Connect and access the platform online here or you can download the app which is available in the Apple App and Google Play stores.

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