In this second of two fall reports on treatment development for Duchenne muscular dystrophy, news about ARM210, CAP-1002, PDE5 inhibitors, drisapersen and CAT-1004 is presented
Update (Feb. 6, 2015): This article has been amended to show that Catabasis' compound CAT-1004 is expected to move into clinical testing in boys with DMD during the first half of 2015.
In a Dec. 2, 2014, webcast for the Duchenne MD community, Sarepta Therapeutics explains plans, answers questions; the presentation is available on Sarepta's site
Cambridge, Mass.-based Sarepta Therapeutics, developer of eteplirsen and other "exon-skipping" drugs designed to treat Duchenne muscular dystrophy (DMD), provided overall reassurance about eteplirsen's development and some details of future plans to the DMD community in a Dec.
Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.
PTC is moving forward with ataluren
Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.
Access to eteplirsen may come in 2015; the company will conduct a confirmatory trial of eteplirsen and two additional trials of the drug
Update (May 27, 2014): Sarepta has acquired a manufacturing facility in Massachusetts to enhance its ability to produce investigational exon-skipping therapies DMD. In a May 22 press release, Sarepta CEO Chris Garabedian said, "While we scale up to address the potential U.S.
The company says it hopes to have feedback from regulators about its experimental Duchenne MD drug drisapersen by the end of June 2014
Update (May 1, 2014): For encouraging results from an open-label, extension study of drisapersen (all participants receive the drug in an open-label study), see Prosensa's May 1, 2014, press release.
Dutch biopharmaceutical company Prosensa outlined plans for drisapersen and other drugs for Duchenne MD in a Jan. 16, 2014, conference presentation and press release
Update (Jan. 21, 2014): Both the Jan. 16, 2014, webcast from the J.P. Morgan conference and the Jan. 21 webcast for patient groups are available on the Prosensa site at Events & Presentations. The patient group webcast recaps many of the same themes as those summarized below from the J.P. Morgan conference presentation.