1. Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

    Posted on August 30, 20135:02 pm

    Aug 30, 2013   Skipping multiple exons at the same time, by using a combination of antisense oligonucleotides, offers the potential to treat a significant number of Duchenne patients. This would address one of the major limitations of current antisense therapy, in that the approach is “personalized” and designed to skip a single exon. Specifically, Read more »

  2. DMD transcript imbalance determines dystrophin levels

    Posted on 3:27 pm

    CureDuchenne’s chief scientific advisor, Dr. Mike Kelly reviewed the recent paper by Annemieke Aartsma-Rus and other researchers.   This is complicated but very important science, we attempt here to explain the findings. http://www.fasebj.org/content/early/2013/08/22/fj.13-232025.abstract The most advanced drugs in clinical development for the treatment of Duchenne (the exon-skipping drug candidates’ drisapersen and eteplirsen as well as the stop-codon read Read more »

  3. Prosensa Provides Updates on Duchenne Development Programs

    Posted on August 14, 20137:31 pm

    On August 13, 2013, Prosensa’s CEO, Hans Schikan and, Chief Medical Officer Dr. Giles Campion,  provided updates on the Company’s development programs for the treatment of Duchenne.  CureDuchenne is pleased to see the progress that Prosensa has made, now with six exon skipping programs at different stages of development. CureDuchenne was the lead organization that Read more »

  4. Sarepta and Prosensa Announcements This Week

    Posted on August 8, 20134:40 pm

    There continues to be good news for the Duchenne community.  We will continue to monitor Duchenne research news and share it with you as it becomes available. Here are three announcements from this week: Sarepta Therapeutics Announces Publication of Eteplirsen Clinical Study Results in the Annals of Neurology Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer Read more »