Pipeline Update: Drisapersen, GSK

Below is the latest update PPMD has received from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit :

To keep you updated on the progress of the drisapersen (previously GSK2402968) clinical development program, as you have requested, I am writing to inform you that the results of the GSK Phase IIb study investigating drisapersen as a potential treatment for DMD boys who have a dystrophin gene mutation amenable to an exon 51 skip will be presented at a scientific conference in the U.S. on the 11th of April 2013.


The Phase IIb results of the DMD114117 study will be presented for the first time at Cold Spring Harbor, RNA and Oligonucleotide Therapeutics, conference (10-13 April 2013). The  abstract title is “Drisapersen treatment for Duchenne muscular dystrophy—Results of a randomized, double-blind, placebo-controlled clinical trial” and will be presented by Dr John Kraus, GSK.

Other recent updates from GSK

Learn more about drisapersen

Drisapersen (previously GSK2402968) consists of short pieces of DNA called “antisense oligonucleotides” or “AONs” that are being tested for their ability to convert deletions near Exon 51 in the dystrophin gene from non-functional “out-of-frame” deletions to more functional “in-frame” deletions, such as those typically seen in boys and men with Becker muscular dystrophy. The strategy is commonly called “exon-skipping”.


Although variations on this strategy might ultimately be used to try to correct deletions in many parts of the dystrophin gene, GSK 2402968 targets the following deletions: 45-50, 47-50, 48-50, 49-50, 50, 52.


 Duchenne Research

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