1. Understand how RNA based therapeutics target the genetic cause of dominant muscular dystrophies, a class of molecules which are already approved for neuromuscular disorders and may be important for dominant muscular dystrophies
  2. Understand our current understanding of the disease mechanism and key clinical features of facioscapulohumeral muscular dystrophy
  3. Understand how key clinical concepts of muscular dystrophies are encapsulated in outcome measures for clinical trials, and how they inform our interpretation of trial results
Session date: 
08/13/2025 - 8:00am to 9:00am CDT
Location: 
UT Southwestern Medical Center
Dallas, TX
United States
See map: Google Maps
Add to calendar: 
  • 1.00 AMA
  • 1.00 Attendance
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