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
Details:
- 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
