exon skipping trial

Drisapersen, designed to treat boys with Duchenne MD with mutations near exon 51 of the dystrophin gene, showed no benefit in a phase 3 trial

posted on September 20, 2013 - 12:55pm
Update (Dec. 19, 2013): In a communication today to patient groups, GlaxoSmithKline (GSK) said that, together with Prosensa, it is continuing to analyze the phase 3 drisapersen trial and other aspects of the drisapersen clinical program, and that it anticipates having results of this analysis in January 2014. Until then, GSK said, dosing of drisapersen in ongoing studies will continue to be...
posted on December 31, 2009 - 1:12am
QUEST Vol. 17, No. 1
Featured in this issue: Duchenne muscular dystrophy exon skipping trial ** Idebenone in DMD ** Gene injections for type 2D limb girdle muscular dystrophy **  Patient registry for LGMD2B and Miyoshi myopathy
posted on March 1, 2008 - 10:21am
QUEST Vol. 15, No. 2
This article includes items about: Duchenne muscular dystrophy, myasthenia gravis, spinal muscular atrophy and acid maltase deficiency (Pompe disease)