Date: September 16, 2025
Time: 11:00 am to 12:30 pm
Room: Dover A - C (Third Floor Level)
Track: Traditional Special Interest Group (SIG)
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.
At the conclusion of the session, attendees should be able to:
This presentation will illustrate advances in therapies for myasthenia gravis, discuss challenges in clinical trials and lessons learned from these and other advances.
In this presentation, Dr. Statland will present updates on his current research into neuromuscular therapeutics.
This presentation will show how Aleeto promise for ALS by protecting motor neurons, improving function, and extending lifespan in preclinical and IIT studies.
In this presentaion, we will discuss GLP-1-related antihyperglycemic medication use is associated with shorter survival in patients with ALS and diabetes mellitus.