We are encouraged by the promising Phase IIb data for eteplirsen, an exon-skipping compound, which we hope could be a breakthrough treatment for Duchenne muscular dystrophy. Currently, there is no treatment for this most common and lethal form of muscular dystrophy. While eteplirsen would likely work in only 15 percent of those with a specific […]
GlaxoSmithKline (GSK) and Prosensa today announced that GSK’s Phase III clinical study of drisapersen, an investigational antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy (DMD) patients with an amenable mutation, did not meet the primary endpoint of a statistically significant improvement in the 6 Minute Walking Distance (6MWD) test compared to placebo. A total […]
CureDuchenne has worked closely with biotech and pharmaceutical companies for the past decade with a goal of accelerating treatments for Duchenne muscular dystrophy.  CureDuchenne was the first organization to partner with a biotech company to develop exon skipping to treat Duchenne almost ten years ago when we invested in Prosensa, a Dutch biotech company.  Since […]
Yesterday, Fergie – a Grammy award-winning singer/songwriter, and new mom! –  joined the ranks of talented and respected celebrities we love.  Over the last few years, Clay Matthews, Ryan Getzlaf, Mack Brown and Scott Niedermayer have spoken out publicly in support of CureDuchenne.  We are very touched by Fergie’s support. Fergie is appearing in a […]
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, […]
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 […]
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 […]
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 […]
  Sarepta Therapeutics announced today plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2014 for the approval of eteplirsen, an exon skipping compound under development for the treatment of Duchenne muscular dystrophy. CureDuchenne, a leader in raising awareness and funding research to […]
  CureDuchenne is pleased that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to drisapersen, an exon-51 skipping compound for the potential treatment of patients with Duchenne muscular dystrophy. Drisapersen is being developed by GlaxoSmithKline (GSK) and licensed from Prosensa Therapeutics. CureDuchenne has worked closely with the FDA to expedite Duchenne drugs […]
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