Gene therapy restored muscle function and prolonged lives in animals with a condition similar to X-linked myotubular myopathy in children

Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital pediatric disease known as X-linked myotubular myopathy. The results, appearing in Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for this devastating disease.