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NMD Pharma’s NMD670 Secures the US FDA’s Orphan Drug Designation to Treat Charcot-Marie-Tooth Disease

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…

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Disease of the Month - Spinal Muscular Atrophy

Disease of the Month – Spinal Muscular Atrophy

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…

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