Investigators: Jeffrey M Statland MD, Rabi Tawil MD
Category: Research - Clinical
The primary goal of this proposal is to hasten therapeutic development for FSHD and improve care delivery by creating a standard clinic-based protocol informed by best practice guidelines to determine the predictive value of clinical characteristics and motor assessments on motor and health outcomes (i.e. need for orthotics, ambulation aides, respiratory support, etc.) – this will help us understand how large a change in outcome assessments would be clinically meaningful, and help us know what baseline FSHD characteristics predict future motor or health outcomes.