The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset
dystrophy, was recently discovered identifying targets for therapy. As multiple drug
companies pursue treatments for FSHD, there is an urgent need to define the clinical trial
strategies which will hasten drug development, including creating disease-relevant outcome
measures and optimizing inclusion criteria. This proposal will develop two new outcome
measures and optimize eligibility criteria by testing 160 patients in 7 sites over a period
of 18 months.