Short research news items about Friedreich's ataxia; Pompe disease; spinal muscular atrophy; and Becker, Duchenne, Emery-Dreifuss, limb-girdle and oculopharyngeal MDs
posted on May 16, 2011 - 2:41pm
Idebenone may help maintain respiratory function in DMD
Santhera Pharmaceuticals announced May 9, 2011, that its drug Catena (generic name idebenone) appears to slow the decline in respiratory function associated with aging in people with Duchenne muscular dystrophy (DMD). Idebenone may improve energy production in muscle and nerve cells.
Promising minigene only partially normalizes heart muscle
posted on January 21, 2009 - 11:14am
A promising “minidystrophin gene” that restores normal muscle force to skeletal and diaphragm muscles in mice with a disease resembling Duchenne muscular dystrophy (DMD) seems to be only partially effective at restoring strength and function to heart muscles.