From 1 to 3 May 2026, the 295th ENMC workshop, “Harmonizing Clinical Monitoring in FSHD: Clinical Outcome Measures, Patient Perspectives, and Digital Innovations for the Upcoming Era of Drug Therapies” took place in Hoofddorp, The Netherlands. The workshop was co-organised by Dr K. Eichinger (USA), Dr E. Bugiardini (UK), and Prof. B. Schoser (Germany).
The workshop aimed to agree on a shared, practical approach to monitoring FSHD in routine clinical care: a way that works in different clinical settings, includes the patient’s own experience, and is ready for the era of new therapies.
Representatives from the FSHD community included Ria de Haas (FSHD Europe), Rajeshri Badiani (FSHD UK), and Amanda Hill (FSHD Society), who contributed by providing valuable patient insights. They gave presentations on patient perspectives on unmet medical needs, including extra-muscular involvement in FSHD, and the role of patient organisations in international collaborations, such as Project Mercury and PaLaDIn.
This collaborative effort is essential for advancing patient care and optimising data collection methodologies, which in turn support robust clinical trials, informed payer decisions (who will pay for medicines, once they are approved), and overall improved outcomes for patients.
Amanda Hill (FSHD Society), Rajeshri Badiani (FSHD UK), and Ria de Haas (FSHD Europe) brought valuable patient insights to the 295th ENMC Workshop, held 1‒3 May 2026.