Clinical results of the gene therapy trial in myotubular myopathy : efficacy on respiratory and motor functions in this extremely severe disease, challenges ahead
In a press release co-signed with the Institute of Myology, Genethon announces that The Lancet Neurology published yesterday the clinical results of a gene therapy trial conducted by Astellas Gene Therapies using a drug candidate developed at Genethon in 24 children suffering from myotubular myopathy, a very severe muscle disease.
