Understanding the Disease Mechanism Behind “Congenital Muscular Dystrophy with Megaconial Myopathy (MDCMC)” to Enable Drug Development for a Functional Rescue.

Time: 11:00 am
day: Preclinical Stream Day 1 AM


  • Importance of understanding and defining disease mechanism and cause for better drug development.
  • Testing different therapeutic strategies with focus on efficacy and ease of administration.
  • Importance of patient voice in therapy