Two Web-based, industry-sponsored presentations that update the Duchenne MD community about exon-skipping drug development will take place Oct. 15 and Oct. 17
Pharmaceutical companies developing exon-skipping drugs for Duchenne muscular dystrophy (DMD) will host Web-based presentations for the DMD community during the week of Oct. 14, 2013.
GSK, Prosensa to present Oct. 15
In a September 2013 presentation, Prosensa CEO Hans Schikan said the company will continue developing exon-skipping drugs for Duchenne MD, despite disappointing drisapersen results
"Our commitment to Duchenne muscular dystrophy remains," said Hans Schikan, CEO of Netherlands-based biotechnology company Prosensa at a Sept. 27, 2013, presentation in New York.
Sarepta Therapeutics plans to target three more dystrophin exons, in addition to exon 51, as potential treatments for Duchenne MD
Biopharmaceutical company Sarepta Therapeutics has announced it will expand the focus of its exon-skipping program for Duchenne muscular dystrophy (DMD) by developing compounds that target exons 45, 50 and 53 of the dystrophin gene, in addition to continuing to develop eteplirsen, which targets exon 51 of this gene.<
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
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.
Dutch biotechnology company Prosensa and multinational pharmaceutical company GlaxoSmithKline are moving ahead with plans to test drugs targeting several exons in the dystrophin gene
Update (Feb. 1, 2013): Dutch biopharmaceutical company Prosensa announced Jan. 29 that it has received orphan drug status in the United States and the European Union for compounds in development for the treatment of Duchenne muscular dystrophy.
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
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
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."