FSHD European Trial Network
In 2021, FSHD Europe established the FSHD European Trial Network (ETN) to support collaboration to reach trial readiness, building on current research on FSHD in Europe.
Developments within the international FSHD field are moving fast. Based on the number of companies developing novel therapies, a drastic increase in the number of trials is expected. This indicates the urgency of trial readiness and underscores the importance of the global initiative Project Mercury in which FSHD Europe is partnering with the FSHD Society.
Performing trials in Europe is challenging because of Europe’s diversity and multilingual situation. Guidelines for clinical trials, pharma regulation and participation, and health care provisions in European countries differ in various subtle ways. Therefore, the FSHD European Trial Network (ETN), has been working on the following aims:
- Increase the commitment of clinicians and researchers in clinical research in Europe;
- Harmonize criteria for clinical and genetic diagnosis, for clinical outcome measures, biomarkers and imaging outcome markers;
- Exchange of clinical experience and genetic reference material;
- Engage Pharma and EMA for a Europe wide collaboration;
- Harmonize treatment and care for all European FSHD patients.
ETN Working groups
As a result, of the ENMC workshops four ETN working groups were initiated. We are very pleased with the steps already taken and work that has been done.
Working group 1: Clinical and genetic diagnosis
Working group 2: Clinical outcome measures
Working group 3: Biomarkers
Working group 4: Imaging outcome measures
The ETN working groups each have a group leader and co-leader and work in close collaboration with the Clinical Research Trial Network, FSHD Society, TREAT-NMD and the European Reference Networks for Rare Diseases.
If you are a clinician or researcher and interested to join one of the working groups, please let us know and reach out to nicol.voermans@radboudumc.nl or ria@fshd-europe.info.
ENMC International Workshops
The European Neuro Muscular Centre (ENMC) has a unique concept of facilitating and organizing workshops initiated by experts in the field of neuromuscular conditions. Several workshops have been organized focusing on FSHD. (Past workshops – ENMC)
During the 268th ENMC workshop (30th September – 2nd, October 2022) 31 experts covering a range of specialties from across Europe, United States and Australia discussed about genetic diagnosis, clinical classification, outcome measures, and biomarkers of FSHD.
In the following workshop, the 265th ENMC workshop on “Muscle Imaging in FSHD: relevance for clinical trials”, 26 participants discussed on the usefulness and harmonization of the imaging techniques specifically in FSHD.
In November 2024, the 279th ENMC workshop ‘Classification, clinical care, outcome measures and biomarkers in childhood onset FSHD: towards standardizing clinical care and ensuring clinical trial readiness.’ will take place, organized by Dr C. Erasmus (The Netherlands), Prof. K. Mathews (USA), K. de Valle (Australia) and Prof. T. Willis (UK).
Join the ETN
We aim to have open membership and to involve experts from all European countries. You might be contacted by clinicians or researchers in your country who are interested or know (future) FSHD experts in European countries not yet involved in the ETN. Please refer them to Ria de Haas (ria@fshd-europe.info).