Workshop reportClinical trial preparedness in facioscapulohumeral muscular dystrophy: Clinical, tissue, and imaging outcome measures 29-30 May 2015, Rochester, New York

- Many national registries now exist facilitating patient access to trials.
- New functional and patient reported outcome measure is being validated.
- More research is required to establish reliable tissue and serum biomarkers.
- Longitudinal muscle MRI studies suggest that imaging is a useful biomarker in FSHD.
- Electrical impedance myography is reliable in certain muscle groups.