ECRA – Towards a Cure for CMT
The Europäische CMT-Forschungsgesellschaft (European CMT Research Association, ECRA) e.V. was officially registered on September 17, 2024, as a non-profit charity organization. ECRA brings together leading researchers from Europe and beyond to advance Charcot-Marie-Tooth research and develop therapies for millions of patients affected by this devastating inherited neuromuscular disease.
This innovative model of structured cooperation among scientists, clinicians, and other health professionals, in collaboration with CMT patient organizations and pharmaceutical industries, is based on the experience of the 1st European CMT Specialists Conference held in Paris in June 2023. This approach promises significant scientific and technological advancements towards curing the various types of CMT.
Together we are stronger. This was the motto of the 1st European CMT Specialists Conference organized by ECMTF in June 2023 in Paris. One of the key outcomes of this conference was the commitment to unite CMT researchers, patient organizations, and industry representatives to make rapid progress in understanding this largely unknown disease. For the first time, relevant stakeholders came together to address the challenges of effective research, including the rarity of the disease, lack of funding, and public awareness. Establishing ECRA was the first step on this path, with a new approach to health data management for CMT to follow.
ECMTF took the initiative to establish a task force of top scientists, clinicians, and young researchers to draft the Articles of Association, leading to the creation of ECRA. After eight online meetings over one year, this objective was finally achieved.
Purpose and Priorities of ECRA
The purpose and priorities of ECRA are defined in its statute as follows:
- Promotion of Innovative CMT Research: ECRA aims to promote innovative CMT research in Europe through close cooperation with patient organizations and industry partners. This includes:
- Encouraging and supporting the development and implementation of joint research projects and conferences, including the introduction of new technologies in CMT research.
- Providing seed capital and expert advice on applying for grants for national, EU, and international research programs.
- Securing long-term funding for CMT research and enabling the development of new therapies by industry, private organizations, and public institutions.
- Establishment of a European CMT Patient Registry: ECRA will initiate and provide technical support for the establishment of a European patient-led CMT patient registry/health database. This includes:
- Developing an adequate infrastructure to collect information and provide access to patients willing to participate in research projects and clinical trials.
- Providing registered patients with up-to-date information on research progress, new drug developments, and clinical trials.
- Ensuring data protection in accordance with the General Data Protection Regulation and developing appropriate ethical guidelines for health data management.
- Promotion of the Next Generation of CMT Professionals: ECRA will promote the next generation of CMT scientists, clinicians, and healthcare professionals by:
- Organizing workshops on CMT-related research topics such as clinical research, pathophysiology, molecular targeting, and therapy development.
- Promoting and supporting the mobility and training of young and advanced scientists, clinicians, and therapists.
- Establishing an ECRA website to report on ECRA’s work, current research results, and publications, and to offer a digital communication system for ECRA members.
- International Collaboration: ECRA will ensure international involvement and collaboration by promoting close cooperation with related scientific and patient organizations such as INC, CMTR, PNS, WMS, ICNMD, ENMC, Treat NMD, ERN Euro-NMD, NORD, EAMDA, and others.
The provisionally elected Executive Board consists of Ingolf Pernice (Vice-President of ECMTF) as interim president and Maike Dohrn (RWTH Aachen University Hospital) as interim vice-president. They will oversee membership recruitment and other business steps to make ECRA operational, including preparing for the first General Assembly, which will elect the first Executive Board and take necessary decisions to get ECRA to work.
The General Assembly is planned for October 24-25, 2025, alongside the 2nd European CMT Specialists Conference in Antwerp. ECMTF and the ECRA taskforce members expect significant interest in this initiative from CMT specialists, patient organizations, politics, and industry, and they hope for generous support from all sides.