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
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...
Weekly injections of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy resulted in a significant difference in walking distance compared to a placebo
The 62-week results from a phase 2b trial of eteplirsen show the drug continued to stabilize walking ability in boys with Duchenne MD
Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at 62 weeks with respect to the distance walked in six minutes.
Investigators in Columbus, Ohio, need to determine the best way to assess thigh-muscle strength in men with BMD before conducting a gene therapy study
A new study to determine the best "outcome measure" (measurable activity) with which to assess thigh-muscle (quadriceps) strength in men with Becker muscular dystrophy (BMD) is seeking participants.
The study, taking place at Nationwide Children's Hospital in Columbus, Ohio, is a necessary prelude to a planned trial of gene therapy involving injections of genes for the follistatin protein in...