Treatment of Inherited Neuromuscular Disorders: Lessons Learned from Ongoing Trials*
Date: September 16, 2025
Time: 11:00 am to 12:30 pm
Track: Traditional Special Interest Group (SIG)
Session Description
Over the past decade, there has been an evolution of genetic testing and discovery of disease mechanisms that have enabled a new era of experimental and approved gene targeted therapy for genetic neuromuscular diseases. Therapeutic strategies have included protein replacement, gene replacement, RNA interference, antisense oligonucleotides, gene editing and small molecule approaches. There is a growing pipeline of investigational therapies for genetic neuromuscular disease and already approved therapy with some real world evidence now available. This session will discuss some of these diseases and therapeutic approaches. It will also cover experience of approved treatments to date including the risks and benefits.
Learning Objectives
At the conclusion of the session, attendees should be able to:
- Discuss the genetic mutations that are associated with the diseases discussed.
- Discuss the available investigational or approved gene target therapy for the diseases discussed.
- Assess the risks/benefits of the new treatments for the diseases/disorders discussed.