GSK2402968

Safety and blood levels of GSK's exon-skipping drug were evaluated in boys with Duchenne MD who were no longer walking and had specific dystrophin mutations

posted on November 2, 2012 - 5:15pm
Update (Sept. 19, 2013): This story was updated to reflect the availability of a scientific paper detailing the trial results; it was published online Sept.

GlaxoSmithKline's Duchenne MD exon-skipping drug moves into a phase 3 trial and is renamed drisapersen; "block skipping" of exons 45-55 in mice looks promising

posted on August 15, 2012 - 2:41pm
Exon skipping is an experimental therapeutic strategy in which regions — exons — of a gene are targeted and blocked ("skipped") by laboratory-designed molecules. The goal is that the remaining genetic instructions will lead to production of a shorter but still-functional protein.

A phase 2 trial of GSK2402968, designed to treat Duchenne MD caused by specific mutations, is now recruiting at 14 sites

posted on June 1, 2012 - 4:05pm
Update (April 9, 2013): The U.S.-based phase 2 trial of drisapersen stopped recruiting new participants in January 2013. Results are expected in early 2014. Update (Aug. 15, 2012): GlaxoSmithKline's exon-skipping drug GSK2402968 has been given a new generic name, "drisapersen."
posted on October 1, 2011 - 10:09am
QUEST Vol. 18, No. 4
Featured in this update: Families, experts meet at BMD ConferenceThree trials study blood-vessel-dilating drugs in BMD, DMDResults of daily, weekly prednisone treatment about the same in DMD

Clinical trials of two different exon-skipping compounds show encouraging results; Duchenne MD participants are being sought for new trials

posted on March 29, 2011 - 11:42am
sClinical trials that use compounds called antisense oligonucleotides to cause skipping of exon 51 of the dystrophin gene in individuals with Duchenne muscular dystrophy (DMD) are moving forward in the United States and elsewhere. Exon skipping for DMD is a strategy that coaxes muscle fibers to ignore, or "skip," the genetic instructions for certain parts of the dystrophin gene so that functional...

A study of exon-skipping drug GSK2402968 in nonambulatory subjects with DMD has opened at Nationwide Children's Hospital in Ohio

posted on June 18, 2010 - 11:58am
Update (Nov. 5, 2012): Results announced in November 2012 showed the drug, now called drisapersen, reached blood levels approximately proportional to the injected dose at two of the three dosage levels tested and was not associated with serious adverse events. See Drisapersen Appears Safe in Non-Walking Boys With DMD.

Investigators presented intriguing data from a trial of an experimental exon skipping drug for Duchenne muscular dystrophy, at the 2010 American Academy of Neurology meeting

posted on April 14, 2010 - 3:21am
The multinational pharmaceutical company GlaxoSmithKline (GSK) and the Dutch biotechnology company Prosensa  announced "intriguing results" following a 12-person trial of an experimental "exon skipping" therapeutic for Duchenne muscular dystrophy (DMD).