Details

Autor(en) / Beteiligte

• Godfrey, J.
• Reuben, E.
• Chandler, F.

Titel

347P Project HERCULES – building an evidence base to inform clinical care and access to medicines in DMD

Ist Teil von

• Neuromuscular disorders : NMD, 2024-10, Vol.43, p.104441, Article 104441.678

Ort / Verlag

Elsevier B.V

Erscheinungsjahr

2024

Quelle

Elsevier ScienceDirect Journals

Beschreibungen/Notizen

• Duchenne UK's Project HERCULES has developed a set of tools and evidence to better understand DMD and inform access to medicines and clinical care as well as further research in this field. This patient-led international research collaboration has included clinicians, academic experts, HTA bodies, patient advocacy groups and pharmaceutical industry partners. It has developed a new disease model describing a transfer stage between ambulatory and non-ambulatory disease stages and providing greater granularity about later stages of the disease. It has also developed a bespoke measure of Health-Related Quality of Life (HRQoL) that captures the impact of DMD, and healthcare interventions, on aspects of HRQoL that matter to those living with DMD. This work now underpins the current priorities of Duchenne UK, informing the DMD Hub, DMD Care, and the Duchenne muscular dystrophy data collection platform. The UK Duchenne data platform, launching in early 2025, will be a crucial tool for engaging with patients and collecting their reported outcomes and perspectives. Duchenne UK's involvement in the PaLaDIn project, funded by EU Horizon 2020 and UKRI, aims to revolutionize neuromuscular disease research and care by integrating registry data, patient-reported outcomes, and wearable data through the Interactium platform, while prioritising robust governance for patient consent and data management. This poster will present these key outcomes and how they are being used in ongoing research as well as exploring how this work can underpin future work in improving clinical care and access to new treatments.