Research Discoveries

1. Gene therapy strengthens weak muscles in congenital myopathy. Click here for more info.

2.  Success of an innovative pre-clinical strategy rescuing myotubular myopathy

 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.