Motor Outcomes to Validate Evaluations in FSHD

Budget

• Three-year project: US$109,715/year 1, US$108,715/year 2, US$108,715/year 3
• FSHD Canada Foundation Contribution to Year 1: US$25,000 (jointly with Friends of FSH)

Lay Abstract

Investigators: Jeffrey Statland, PI (University of Kansas Medical Center); and Rabi Tawil, Co-PI

(University of Rochester Medical Center)

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.