Story includes research items about: centronuclear myopathy; Duchenne, limb-girdle, and Emery-Dreifuss muscular dystrophies; and spinal muscular atrophy.
Gene therapy — inserting new genes into cells to treat disease — first became a reality in the early 1990s. Now past its earliest years and into its adolescence, the science has progressed along two major pathways.
The first seeks to define the best way of delivering genes inside the shell of a defanged virus, with the precise type of viral shell dependent on the target tissue. Viral delivery...
In this article: research news about prednisone in Duchenne MD; tests for Duchenne and Becker MDs; and the latest progress in research for spinal muscular atrophy