Duchenne Map: Connecting all stakeholders in the global dystrophinopathy community to advance research, therapy development, and healthcare decision-making

    Principal Author: Katerina Tzima


    Fragmentation, duplication of effort, and lack of reliable information are problems that all rare disease communities face. These factors have a direct effect on the quality of life and care of people living with dystrophinopathies and their families, but also impact access to treatment, and recruitment to clinical trials, eventually negatively affecting the development of new medicines.

    The Duchenne Data Foundation has developed the Duchenne Map, to make relevant information accessible and connect patients with their local patient organizations, healthcare providers, care centers, researchers, research institutions, and pharmaceutical companies. Data is stored in the Duchenne Data Repository following a user-centric data governance and GDPR-compliant framework. Users register on the Map and complete their profile. A double validation process assures the authenticity of the user and the information displayed. Once registered and validated, users can use the Map to find relevant information on Patient Organizations, Pharmaceutical Companies, and Clinical Trials.

    Currently, registered users can find:

    – 52 Patient Organizations, 5 Pharmaceutical Companies, 137 Clinical Trials

    – A personalized clinical trial filter function. Users can search for recruiting clinical trials in their area according to age, corticosteroid use, and ambulation status.

    Additional filters allowing to search for clinical trials based on the mutation type and find care centers offering specific services are currently under development.

    Registration campaigns are ongoing to spread awareness of the functionality of the Map. Outreach activities are planned to link the Map with other data platforms in the dystrophinopathy community.

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