NMD Pharma recently published data demonstrating that their drug candidate, NMD670, improves muscle function and neuromuscular transmission deficits in both animal models and patients. Dr. William David Arnold, a member of the CMTRF Scientific Advisory Board, who was part of the international project, comments on the importance of these findings for the CMT community:
“NMD Pharma has uncovered a promising treatment approach to enhance neuromuscular junction transmission. Serving as the interface between the nervous system and muscles, the neuromuscular junction is vital for motor function. Mounting evidence suggests that disruptions in neuromuscular junction transmission are common in various neuromuscular disorders, including CMT. Hence, theoretically, this treatment method holds potential for improving muscle function in individuals with CMT. Importantly, it may have relevance across different types of CMT, irrespective of the specific gene mutation responsible for the condition.”

“This new data coming out of NMD Pharma is extremely exciting for the CMT community,” adds Dr. Riann Egusquiza, Director of Research for CMTRF. “This evidence of translatability from animal models to patients is a huge accomplishment and provides a lot of confidence that this compound could provide a treatment option for CMT patients.”