We wanted to let you know that Sarepta Therapeutics will host a webcast and conference call on October 17.  Please see information below from Sarepta.  We encourage you to submit your questions to Alicia@cureduchenne.org by Monday morning.  We will collect and forward them to Sarepta prior to the webcast.  This is a great opportunity to […]
  GSK and Prosensa will hold a joint webinar on 15th October at 16:00 – 17:30 GMT (17:00 – 18:30 CET, 12:00 – 13:30 EST, 09:00 – 10:30 PST). Following on from the disappointing results of the phase III DMD114044 study which did not meet its primary or key secondary endpoints, GSK will share the […]
WMS 2013 was full of excitement and interest as results of current clinical trials and early stage research were discussed.  Here are some highlights, thank you Abby Bronson for summarizing the highlights.  ORAL PRESENTATIONS Dr. Elizabeth McNally of the University of Chicago discussed her work focusing on identifying genetic modifiers that cause different phenotypes in […]
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 […]
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