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
The company plans to submit a New Drug Application to the U.S. Food and Drug Administration later this year, and has committed to conducting two confirmatory post-approval clinical trials
Dutch biopharmaceutical company Prosensa has outlined plans to seek accelerated approval in the United States for its experimental drug drisapersen, under development for the treatment of Duchenne muscular dystrophy (DMD). The company will seek approval for the drug in Europe as well.
In a May 22, 2014, letter, Prosensa provides updates on drisapersen and other Duchenne MD compounds in its pipeline, and on its natural history study
Dutch biopharmaceutical company Prosensa, developer of the experimental Duchenne muscular dystrophy (DMD) drug drisapersen, says it is moving the compound forward through regulatory agencies in the U.S. and Europe and will resume administering it to a first group of trial participants in the third quarter of 2014.
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.
A phase 2 trial of Duchenne MD drug drisapersen found the high-dose group walked farther than the placebo group at 24 and 48 weeks
Update March 27, 2014: Prosensa and United Parent Projects Muscular Dystrophy jointly presented an educational webinar for patients and families affected by Duchenne muscular dystrophy on March 25, 2014. The approximately one-hour presentation is archived at Events & Presentations on the Prosensa site and includes a thorough discussion of Prosensa's plans for drisapersen and other exon-...
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.
Drisapersen developer Prosensa will update the investor community via Jan. 16 webcast and the DMD patient community via Jan. 21 webcast
Dutch biopharmaceutical company Prosensa, developer of the experimental drug drisapersen and other experimental compounds for Duchenne muscular dystrophy (DMD), will update the investor community via a Thursday, Jan. 16, 2014, presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation will be webcast at 10 a.m. Pacific time/1 p.m. Eastern time.
On Tuesday, Jan. 21,...