Mizzou’s bench-to-bedside efforts support a new drug for a neuromuscular disorder with no cure

By Brian Consiglio

April 14, 2026
Contact: Brian Consiglio, [email protected]
Photo by Ben Stewart

The University of Missouri is at the forefront of developing a novel treatment to improve muscle strength and functioning for patients with Charcot-Marie-Tooth (CMT) disease, the most common inherited neuromuscular disorder, for which no cure currently exists.

W. David Arnold, the executive director of the NextGen Precision Health initiative, presented encouraging top-line results from a recent clinical trial of the novel drug candidate ignaseclant during a late-breaking session at the Muscular Dystrophy Association Conference in Orlando. Arnold was the senior author on an earlier study, which laid the scientific foundation for the work to progress to humans.

"In a multicenter clinical trial conducted at NextGen and 18 additional international sites, participants with CMT who received ignaseclant demonstrated improvements in muscle strength and functional task performance," Arnold said. "Notably, after 21 days of treatment, participants were reassessed seven days after stopping the drug, and the benefits were maintained, or even further improved, during that period."

"Since there is no available treatment for CMT, there is a huge need for therapies in the space," Arnold said. "We enrolled patients in our trial very quickly, which shows we are well-positioned to help meet the demand, and we are excited that the drug seems to help people."

The success of these clinical trials further strengthens Mizzou's reputation as a leader in neuromuscular disease research and highlights its strength in translating early, foundational discoveries into clinical advances that ultimately improve patients' lives.

With Mizzou's world-class investigators working in the Roy Blunt NextGen Precision Health building, a state-of-the-art facility with research and clinical space under one roof, this translational work happens faster and more efficiently than ever before.

Excited muscles

Arnold, a professor in the School of Medicine and physician at MU Health Care, began collaborating with Thomas Holm Pedersen, cofounder of NMD Pharma, the developer of ignaseclant, in 2019. The goal of this collaboration was to understand how impairments in nerve-to-muscle communication that drive muscle contraction could be targeted to improve function in patients with neuromuscular disorders.

One potential target is the CIC-1 ion channel, a protein in muscle cells that helps stabilize the muscle membrane and prevents it from becoming too easily activated.

In neuromuscular diseases such as CMT, the signals sent from nerves to muscles are too weak to effectively trigger contraction. By partially inhibiting CIC-1, muscle fibers become more responsive to signals. This increased sensitivity can amplify nerve input and ultimately improve muscle force generation.

In addition to CMT, Arnold is hopeful that ignaseclant may benefit patients with other neuromuscular diseases, including spinal muscular atrophy (SMA) type 3 and myasthenia gravis (MG). Multicenter clinical trials are underway at sites internationally, including Mizzou, to determine whether the drug provides similar benefits in these conditions.

"What's exciting about this drug is its potential to benefit all forms of CMT, which are linked to more than 100 different genetic causes," Arnold said. "By enhancing the signals nerves send to muscles, this approach may be applicable across a range of neuromuscular diseases, regardless of the underlying mutation. Seeing this work progress from foundational preclinical research on the first floor of the NextGen building to a human clinical trial on the third floor is incredibly rewarding."

The effort reflects a team science approach, with contributions from Ryan Castoro, Kris Kelly, Jose Viteri, Nathan Kerr, Vaibhav Oberoi, Arsh Ketabforoush, Meifang Wang and Mizzou's Clinical and Translational Science Unit.

"We are proud to advance a seamless bench-to-bedside pipeline that accelerates the delivery of new therapies to patients," Arnold said.

Arnold, an avid runner and swimmer who's competed in multiple marathons and triathlons, feels a deep motivation for his work.

"I love movement, which inspired me to want to help those whose movement has been limited or taken away from them," Arnold said. "To see patients in this clinical trial show positive improvements with their symptoms is so gratifying. It doesn't get better than that."