Dr. Adeline Vulin from Nationwide Children’s Hospital presented the latest data from her work in Dr. Kevin Flanigan’s lab on the new duplication mouse model and the results of exon skipping experiments in that mouse. Duplications account for approximately 6% of all Duchenne mutations, and duplications on exon 2 account for 10% of those mutations.  The […]
Here are top line notes from the PRO044 presentation at World Muscle Society, Oct 4. Age of inclusion:  5-16 Mean age:  9 The drug was well tolerated. They saw trace dystrophin and reverant fibers pre- treatment. 18 subjects, 9 cohorts… SubQ delivery: .5mg/kg, 1.5mg/kg, 5mg/kg, 8mg/kg, 10mg/kg, 12mg/kg IV delivery:  1.5mg/kg, 5mg/kg, 12mg/kg 5 weekly […]
Conference call and live webcast scheduled for 11:00 a.m. ET (5:00 p.m. CET)  Tuesday, October 8, 2013 LEIDEN, The Netherlands – October 3, 2013 – Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, will hold a conference call on Tuesday, October 8, 2013 […]
We wanted to let Duchenne families know about a new online resource on exon skipping from Sarepta Therapeutics.   Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced a new online resource center, called Let’s Skip Ahead, for families affected by Duchenne muscular dystrophy (DMD) and their healthcare providers. The new website, […]
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 […]
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