NMD Pharma Initiates ESTABLISH Study in Patients with Charcot-Marie-Tooth Disease
Aarhus University and The Ohio State University are world-leading institutions in neuromuscular disease research.
- Aarhus University and The Ohio State University are world-leading institutions in neuromuscular disease research.
- The primary aim of this study is to discover whether patients with CMT have neuromuscular junction (NMJ) transmission deficits.
- The hypothesis for the study is that if patients with CMT are found to have deficits in NMJ transmission, then the novel treatment approach being developed by NMD Pharma could potentially have beneficial clinical effects on neuromuscular performance in these patients.
- NMD Pharma is developing first-in-class small molecule inhibitors of the muscle specific chloride ion channel, the ClC-1 ion channel.