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16 - 02 - 2017

Treatment of Early Childhood Myopathy

Myotubular myopathy causes severe muscle deficiency frequently followed by fatal respiratory failure in young children. The feasibility of gene therapy for this disease was demonstrated in 2014 in mice and dogs by Genethon, the AFM-Telethon laboratory, in collaboration with two American universities.

A new study in dogs, that aims to determine the doses and test injection modalities compatible with clinical trials has just been published. The treatment was well tolerated and greatly extended the life expectancy of the animals and corrected the muscle weakness. These results are encouraging enough to move to a clinical trial.