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. via Health News from Medical News Today Read More Here..

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