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The CMTA Is Accelerating Research Through Gene Therapy
…two mouse models of CMT: CMT1X and CMT4C. He is also extending his efforts to develop novel therapies for CMT1A. Dr. Harper has worked extensively in muscular dystrophy and is…
CMTA Seed Money Draws NIH Support For Type 2 Gene Editing
…100 different gene mutations causing CMT. Most people (90%) have one of four types of CMT: CMT 1A (PMP22); CMT 1B (MPZ); CMT 2A (MFN2) and CMT 1X (GJB1). It…
The CMTA Mourns the Loss of Scientific Advisory Board Member and Esteemed CMT Clinical Researcher
…the understanding of CMT, and moving us closer to achieving the CMTA’s vision of a world without CMT. She was highly collaborative and worked with a wide variety of researchers….
CMT4 Research
…to advance research for CMT. Support CMT Research View the projects currently active or recently completed in the CMT4 drug development pipeline. CMT4 RESEARCH PROJECTS CMT4A Research CMT Type 4A…
Alliance Partner NMD Pharma A/S Launches Phase II SYNAPSE-CMT Trial with First Patient Dosed at CMTA Center of Excellence
The Charcot-Marie-Tooth Association (CMTA) congratulates CMTA-STAR Alliance Partner NMD Pharma A/S (NMD Pharma) on initiating its Phase II SYNAPSE-CMT trial. This significant milestone reflects a shared commitment to advancing therapeutic…