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.
Idebenone failed to show a significant difference when compared to a placebo in Friedreich's ataxia
posted on May 22, 2009 - 3:01pm
On May 19, 2009, Santhera Pharmaceuticals reported that a phase 3 trial of its idebenone compound Catena showed the drug was not associated with a statistically significant benefit in 70 children between 8 and 17 years old with Friedreich's ataxia who took it for six months. (See the company's press release, Santhera's US Phase III IONIA Trial in Friedreich's Ataxia Misses Primary Endpoint.)