Kick-Off Panel Discussion: Dissecting Fundamental Questions Arising for Each Therapeutic Approach
Time: 9:05 am
day: Day One
• A critical overview of the current status of knowledge and therapies for neuromuscular disorders, focusing on ALS, SMA and DMD
• What have we unravelled over the last year that will drive more effective clinical development to reach patients in need?
• What are the unique challenges for ASO therapy, gene therapy, gene modification and small molecules
• How can we solve the current clinical development challenges such as demonstrating efficacy and effectiveness to quickly realise true victory for patients?
• What do we need to do as community to accelerate the discovery and development of meaningful treatments?
• Different therapies for the same disease, what should we be looking to learn and compare and contrast with these different approaches in the next few years?