Drisapersen-treated trial participants with Duchenne MD had higher levels of dystrophin protein in their muscle samples at 25 weeks than did placebo-treated participants
posted on August 21, 2013 - 12:17pm
Multinational pharmaceutical company GlaxoSmithKline (GSK) has released encouraging results about dystrophin protein production in a phase 2, non-U.S. trial of its exon-skipping compound drisapersen in boys with Duchenne muscular dystrophy (DMD).
The drug gentamicin increased dystrophin levels in boys with DMD due to a nonsense mutation, but didn't improve strength at the doses used
posted on April 14, 2010 - 3:34am
Results of the MDA-supported trial of gentamicin in Duchenne muscular dystrophy (DMD) were presented Wednesday, April 14, at the annual meeting of the American Academy of Neurology (AAN), held in Toronto.
Levels of the needed dystrophin protein increased in six out of 12 participants who received the drug for six months. No functional improvements were seen.