The role of neuromuscular transmission failure and ClC-1 inhibition as a novel therapeutic approach across neuromuscular diseases; positive proof of mechanism in patients with myasthenia gravis

Date/Time: Sunday, September 10, 2023 - 5:15 PM – 6:15 PM
Track: Satellite Symposia
Room: Franklin Hall 1 (4th Floor)
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Sponsored by NMD Pharma

In a group of neuromuscular diseases, dysfunctions that cause excitation failures of muscle fiber action potential at the neuromuscular junction (NMJ) lead to excessive and, in some cases, life-threatening muscle weakness. Multiple cellular mechanisms can cause NMJ transmission failure in these diseases, commonly affecting the release of acetylcholine from pre-synaptic nerve terminals or the post-synaptic ability of muscle fibers to respond to that release.
During this symposium, we will examine how different neuromuscular diseases affect the NMJ transmission including spinal muscular atrophy, Charcot-Marie Tooth and myasthenia gravis. We will review current clinical evidence of these abnormalities in patients, their correlation with the severity of their symptoms and the potential of skeletal muscle-specific ClC-1 ion channels inhibition to correct these deficits.
Unpublished data results from a proof of mechanism study utilizing a novel and first in class ClC-1 inhibitor in patients with myasthenia gravis will be presented and discussed; patients enrolled in a randomized, double-blind, placebo-controlled, three-way crossover study received a ClC-1 inhibitor and demonstrated clinically relevant improvements.


W. David Arnold, MD

Exec. Dir., NextGen Precision Health initiative, University of Missouri

Thomas H. Pedersen, PhD

Founder and Chief Executive Officer, NMD Pharma

Jorge A. Quiroz, MD, MBA

Chief Medical Officer, NMD Pharma