Program Introduction
Delve into the cutting-edge advancements in Facioscapulohumeral Muscular Dystrophy (FSHD) alongside the dynamic duo of Dr. Leo H. Wang and Dr. Maya N. Hatch in this symposium recorded at the 2024 MDA Clinical & Scientific Conference Summit. Positioned as the third most prevalent muscular dystrophy, FSHD stands as a genetic enigma with intricate roots, showcasing a progressive saga of asymmetrical muscle weakness. Initially targeting the facial and shoulder muscles before spreading its grip onto the extremities, this disorder's clinical narrative presents a formidable challenge in quantifying outcomes. It's an imperative moment to unearth dependable and sensitive metrics for clinical trials, navigating the labyrinthine landscape of FSHD. Acquire invaluable insights into mastering daily functionalities, tackling complications head-on, and remain abreast of the latest breakthroughs in establishing rock-solid measures for gauging progress.
Learning Objectives
- Evaluate the use of RWS as an outcome measure for FSHD clinical trials and translate changes in RWS to clinically meaningful outcomes
- Discuss the mechanism of FSHD disease that leads to challenges in DUX4 biomarker detection
Available Credit
- 1.00 AMA PRA Category 1 CreditTM
- 1.00 Participation