Shots:The US FDA has granted ODD to NMD670 for treating Charcot-Marie-Tooth disease (CMT). It is small molecule that inhibits CIC-1 to enhances muscle responsiveness, improving neuromuscular transmission & restoring function
NMD670 (PO, BID) is under P-IIa (SYNAPSE-CMT) evaluation for its efficacy, safety & tolerability in adults (n=80) with CMT1 or CMT2 subtype for over…

Shots:To keep our readers acquainted with several disease conditions, ongoing trials, and available treatment options, PharmaShots brings every month a detailed take on a particular disease after thorough research Continuing the series for the disease of the month, PharmaShots brings this month a summary of Spinal Muscular Atrophy, a genetic neuromuscular disease that affects…