1. Gene therapy strengthens weak muscles in congenital myopathy. Click here for more info.
Using an innovative strategy, researchers at the IGBMC (France) managed to cure animals affected by myotubular myopathy, also called X-linked centronuclear myopathy. Through a gene modulation approach, J. Laporte’s team observed disappearance of clinical signs of this rare disease and a full rescue of lifespan. In addition to the therapeutic potential, these results shed a new light on the mechanisms involved in Centronuclear myopathies. Click here for press release.