This CME activity took place on Thursday, October 16, 2024, and is eligible for AMA PRA Category 1 Credits™ only to those who attended the activity virtually on this date.
Program Introduction
Join Leo Wang and Maya Hatch for an insightful journey into the world of Facioscapulohumeral Muscle Dystrophy (FSHD), covering its etiology, clinical presentations, and the complexities of assessing treatment outcomes in clinical trials. Key focuses include the innovative Reachable Workspace (RWS) metric for functional assessment, the impact of current clinical trials, and emerging therapeutic targets. Discussions will also highlight biomarker insights, disease mechanisms like DUX4 regulation, and future strategies for FSHD management. Don’t miss this opportunity to gain insights from experts shaping the future of FSHD research and care, along with perspective from a real-world patient living with FSHD.
Learning Objectives
Evaluate the use of RWS as an outcome measure for FSHD clinical trials to measure disease progression, impact on ADLs, and effect of therapy
Discuss the mechanism of FSHD disease that leads to challenges in DUX4 biomarker detection