Thomas Holm Pederson, NMD Pharma - CMT Summit Keynote 2025
Автор: HNFoundation
Загружено: 2025-09-19
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At the 2025 CMT Summit in Nashville, we had the privilege of welcoming NMD Therapeutics as a keynote speaker. NMD shared their revolutionary approach to treating neuromuscular diseases like Charcot-Marie-Tooth (CMT), focusing not just on nerves but directly on improving muscle function.
Their lead compound, NMD670, targets a skeletal muscle-specific chloride channel (CLC1) that normally acts as an electrical brake. In diseases like CMT, this brake can interfere with proper muscle activation. By selectively inhibiting this channel, NMD670 enhanced the ability of weakened neuromuscular connections to activate muscle fibers, offering a powerful new pathway for improving strength and mobility.
During the keynote, NMD presented promising results from both animal models and human trials. In patients with myasthenia gravis, a single dose of NMD670 improved hand strength, speech clarity, and eye control. Even more compelling, repeated dosing appeared to support nerve regrowth and reconnection to muscle fibers, hinting at disease-modifying potential.
NMD Therapeutics is currently conducting Phase 2 trials in three conditions, including CMT, and shared insights into how neuromuscular junction dysfunction directly correlates with functional challenges in CMT patients. They also invited attendees to learn more about their ongoing clinical trial aimed at delivering the first targeted treatment for the neuromuscular connection in CMT.
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