DMD Expanded Core Dataset
Recognising a need to update and develop the existing Duchenne muscular dystrophy (DMD) data set collected by TREAT-NMD affiliated registries, a working group of key opinion leaders, patient representatives & registry curators from around the world was established in 2019. This group met at a workshop in Amsterdam in June 2019 to reach initial consensus on an expanded core dataset (ECD). After subsequent additional feedback, Version 1 of the ECD was agreed in April 2020 ready for testing. Meanwhile, funding for this project has been agreed, a project team is now recruited and 13 registries from Europe, the US and Canada have joined a pilot exercise to carry out this testing. The pilot will undertake to:
- Perform a gap analysis of the ECD vs the data set currently collected in each pilot registry
- Identify challenges of integrating the ECD into existing platforms
- Establish the feasibility of data collection for each data item in a clinical or a patient setting
Alongside this, a new TREAT-NMD Universal Registries Platform (URP) will be developed, specifically designed for DMD registries to collect the ECD, which in turn, will lead to a more efficient mechanism of data collection and analysis, for registry enquires – even where these are of increasing complexity. A number of registries in the pilot project will undertake beta-testing of this new URP. For further information on this or any other aspect of the project, please contact the Project Manager: john.mckenna@TREAT-NMD.com
