The 2nd European Charcot-Marie-Tooth (CMT) Specialists Conference, held in Antwerp, Belgium, from October 23-25, 2025, successfully brought together 136 leading scientists, clinicians, patients, and industry partners from around the globe. In a follow-up webinar on November 14, 2025, key organizers summarized the conference’s groundbreaking discussions and charted a course for the future of CMT research.
The event was a collaborative effort by the European CMT Federation (ECMTF), the University of Antwerp, and the European CMT Research Association (ECRA). It featured a supportive video message from EU Commissioner for Health, Olivér Várhelyi, and was co-funded by the EU4Health program.
Key Takeaways and Scientific Highlights
During the follow-up webinar, moderated by Dr. Maike Dohrn (University Hospital Aachen), the newly elected ECRA president, Prof. Vincent Timmerman (University of Antwerp), outlined the conference’s primary goals. These included increasing awareness of CMT, creating strategic care guidelines, engaging the pharmaceutical industry, developing data-sharing frameworks, and firmly establishing patients as partners in research.
Dr. Dohrn summarized the dense scientific content, highlighting several key areas of progress:
- New Genetic Insights: Novel knowledge was shared on genes like DAS 2 and NAS one, as well as SORD2P inversions and trinucleotide expansions.
- Advanced Disease Models: Discussions covered innovative models such as IPC derived assembloids that mimic nerve cell environments and new CMT1A mouse models to understand disease mechanisms.
- Therapeutic Concepts: An overview of genetic and gene therapies was presented by Prof. Kleopas Kleopa, alongside novel research on A1 antitrypsin and ADAC 6 inhibitors.
- Biomarkers and AI: The importance of circulating blood biomarkers for CMT1A and the role of Artificial Intelligence in differentiating CMT from other neuropathies were key topics.
- Patient-Led Initiatives: The conference showcased networks like ACMT RET, a patient-led network, and tools for movement analysis to better define patient journeys.
A Unique “Patients as Partners” Format
Prof. Ingolf Pernice, the founding president of ECRA, emphasized the conference’s unique format. Unlike larger scientific meetings, the comparatively small size of the Antwerp conference was a key asset, fostering an atmosphere of open, collaborative discussion and strategic coordination among all stakeholders. This created what one survey respondent called a “unique format” that delivered major value through abundant opportunities for networking and mingling.
The core theme was a paradigm shift toward viewing “patients as partners.” The event highlighted that patients are not just subjects of research but active contributors who can make a difference. The need for empathy, education, and better access to specialists, potentially through digital care and telemedicine, were central themes.
Future Direction and the Role of ECRA
A major outcome of the conference was the election of a new, enlarged ECRA board, with Prof. Timmerman succeeding Prof. Pernice as president. ECRA will focus on mentoring young researchers, supporting joint projects, and seeking funding.
The organizers confirmed that the conference will not be an annual event due to the significant cost and effort involved. Instead, a biennial schedule is anticipated, allowing time for meaningful progress between meetings. In the interim, the community is encouraged to engage with the Peripheral Nerve Society (PNS) meetings.
The importance of the in-person format was a recurring point in the webinar. Prof. Timmerman stressed that face-to-face interactions, poster sessions, and informal dinners are irreplaceable for building the strong, collaborative relationships needed to drive research forward. This sentiment was echoed by attendees, who praised the “very special” and “communicative” atmosphere. Videos from the conference are available on the ECMTF YouTube channel to provide a wider audience with an impression of the event.
