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Charcot-Marie-Tooth Disease: Breakthroughs to a Cure
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Scientists at Ionis Pharmaceuticals, in collaboration with the Charcot-Marie-Tooth Association (CMTA), have identified a promising early-stage therapeutic approach for CMT Type 1A (CMT1A), the most common form of the disease, accounting for half of all cases. There is currently no treatment or cure for CMT, making the CMT1A research a significant and hopeful breakthrough for medical researchers, healthcare professionals, patients and families.

Overview

 

Charcot–Marie–Tooth disease (CMT), is a genetically and clinically heterogeneous group of inherited disorders of the peripheral nervous system characterised by progressive loss of muscle tissue and touch sensation across various parts of the body. Currently incurable, this disease is the most commonly inherited neurological disorder, and affects approximately 1 in 2,500 people equating to approximately 26,000 people in the United Kingdom and 128,000 people in the United States. CMT was previously classified as a subtype of muscular dystrophy.

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