ECRA was founded to give CMT research in Europe new momentum. It is a project of the European CMT Federation (ECMTF) and is based upon the works and a decision of the “1st European CMT Specialists Conference” held in June 2023 in Paris. We aim to leverage the diverse group of Charcot-Marie-Tooth (CMT) diseases as a model to create an urgently needed forum for EU-wide coordination to confront key roadblocks shared across the iNMD field, and rare genetic diseases in general. 

 

ECRA is a non-profit association of key stakeholders across disciplines: scientists, clinicians and other health professionals, representatives of European patient organizations, pharmaceutical industries and policymakers. Recent progress in diagnostics, therapies, and outcome assessment will be contextualized within the unique practical challenges relating to rare diseases: lack of knowledge and awareness, difficulty for patients to find a specialist within a reasonable distance, fragmentation and inaccessibility of relevant data for researchers, industry’s reluctance to invest in research etc. 

 

ECRA is made to attack these issues, to boost research in CMT, to develop new approaches for genetic analysis and therapies, explore AI applications in diagnostics and for therapy, establish a system of digital care bridging geographical distance between patients and specialists, spread information and foster education, make relevant health data available to scientists in conformity with the GDPR and EHDS, and invite industry to engage in joint projects paving the way towards developing a cure for the many subtypes of CMT.