December 6, 2017
A paper published December 4th on the Journal of Clinical Investigation website reveals an exciting potential treatment for patients with Charcot-Marie-Tooth, the progressive neuromuscular disease that affects more than 2.8 million people worldwide. Scientists at Ionis Pharmaceuticals, in collaboration with the Charcot-Marie-Tooth Association, have identified a promising early-stage therapeutic approach with antisense technology for CMT Type 1A, the most common form of the disease, accounting for half of all cases. There is currently no treatment or cure for Charcot-Marie-Tooth, making the research a significant and hopeful breakthrough for medical researchers, healthcare professionals, patients, and families.
Charcot-Marie-Tooth disease type 1
Charcot-Marie-Tooth disease type 1A