From 1–3 May 2026, representatives from Project Mercury participated in the 295th ENMC International Workshop on “Harmonizing Clinical Monitoring in FSHD”, held in Hoofddorp, The Netherlands. The workshop brought together leading clinicians, physical therapists, outcomes researchers, industry representatives, and patient organizations with a shared objective: to move toward global consensus on what data should be collected during routine clinical visits for people living with facioscapulohumeral muscular dystrophy (FSHD).
Why this workshop matters
As FSHD enters a new era of therapeutic development, the lack of harmonized clinical monitoring has become a critical bottleneck. Differences in outcome measures, visit structures, and data standards across countries limit the comparability of registries, slow clinical trial readiness, and complicate downstream health technology assessment (HTA) and payer decision‑making.
The ENMC workshop directly addressed these challenges by focusing on:
- Alignment of clinical outcome measures used in routine care,
- Integration of patient‑reported outcomes and digital monitoring tools,
- Improved patient stratification across genetic and clinical phenotypes,
- Identification of unmet clinical needs, including extra‑muscular involvement and fatigue.
Representing the patient voice: Project Mercury’s contribution
Amanda Hill was invited to present during Session 5: PaLaDin Consortium and Patient Organisations, highlighting the initiative’s goals, progress, and relevance to the workshop’s objectives.
During the presentation, Project Mercury shared:
- Progress achieved over the past three years in building a patient‑centered framework for clinical data collection,
- Insights from patient engagement on which outcomes matter most in daily life,
- How patient‑driven priorities can guide decisions about minimum data sets in clinical practice and registries.
This perspective resonated strongly with the workshop’s broader goal of ensuring that harmonization efforts do not merely standardize data for scientific convenience, but also reflect outcomes that are meaningful to people living with FSHD.
Alignment with FSHD Europe and broader patient initiatives
The workshop also featured a complementary presentation by Ria de Haas, representing FSHD Europe, who outlined:
- The development of a patient journey map,
- New patient support toolkits,
- An emerging HTA disease progression model for FSHD.
Together, these contributions underscored a shared commitment across patient organizations to align lived experience, clinical monitoring, and policy‑relevant evidence generation. By engaging early in consensus‑building with clinicians and researchers, Project Mercury positions patient advocacy not as an external voice, but as an integral contributor to clinical system design.
Looking ahead
For Project Mercury, participation in the 295th ENMC workshop marks an important milestone: a clear signal that patient‑centered frameworks are not only compatible with scientific rigor, but essential to the future of FSHD care, research, and access to therapies.
