BiostockNMD Pharma Reports Topline Phase IIa Results
NMD Pharma announced data from the SYNAPSE-CMT study evaluating ignaseclant in Charcot-Marie-Tooth disease types 1 and 2. The CIC-1 inhibitor showed consistent functional improvements regarding muscle strength, motor performance, and patient-reported outcomes. Ignaseclant targets the skeletal muscle-specific chloride ion channel 1.