1. Best Practices in Clinical Study Design for Rare Diseases

    Posted on April 30, 201310:02 pm

    I am leaving Washington DC after attending the Best Practices in Clinical Study Design for Rare Diseases symposium organized by Ed Conor and Abby Bronson at Children’s National Medical Center.  The FDA and NIH were collaborators in this symposium and I was honored to be a part of one of the four roundtable panels. Two Read more »

  2. GSK Update on Extension Study for Disapersen

    Posted on April 24, 201312:33 pm

    The purpose of this communication is provide an update to you, as you have requested, on GSK’s plans for providing the option of continued access to drisapersen for boys completing the US Ph IIb 48 week study (DMD114876) – clinicaltrials.gov link: http://clinicaltrials.gov/ct2/show/NCT01462292?term=duchenne+muscular+dystrophy&rank=52 GSK is committed to providing boys who have completed the clinical studies with Read more »

  3. GSK Drisapersen Notes from the MDA Conference

    Posted on April 23, 201310:12 pm

    Dr. Thomas Voit presented the current data from GSK’s drisapersen’s phase ll study at the MDA Conference in Washington DC.  There was one new slide presented since the Cold Springs Harbor meeting. The continuous treatment arm (n=18) showed a clinically meaningful and statistically significant difference from placebo (n=18) on 6MWD. A new slide was shown:  The 4 Stair Read more »

  4. Eteplirsen Notes from the MDA Conference

    Posted on 8:49 pm

    This afternoon Sarepta presented data on their trial for their lead candidates to skip exon 51 in Duchenne muscular dystrophy at the MDA Conference in Washington DC.  Most of this has been presented recently, there are a few more weeks of data here. Dr. Jerry Mendell presented the 74 week data for eteplirsen: Eteplirsen continues to Read more »


    Posted on April 22, 20135:29 pm

    The Fiscal Year 2013 (FY13) Defense Appropriations Act provides for $3.2 million (M) to the Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP) to support innovative, high-impact Duchenne muscular dystrophy research. This program is administered by the US Army Medical Research and Materiel Command through the Office of the Congressionally Directed Medical Research Programs Read more »

  6. Sarepta Therapeutics Announces FDA Will Consider Accelerated Approval for Eteplirsen After Further Review of Data on Dystrophin and Clinical Outcomes

    Posted on April 15, 201311:12 pm

    Eteplirsen Manufacturing and Clinical Activities Continue as Planned CAMBRIDGE, MA–(Marketwired – April 15, 2013) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT) today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding a potential application for accelerated approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). The FDA has Read more »

  7. Drisapersen, Etiplersen, GSK, Prosensa and Sarepta, a Positive Week for Duchenne

    Posted on April 11, 201311:09 pm

    Thanks to GSK, Prosensa and Sarepta and, the landscape is changing for the patients and families that are suffering from Duchenne, a severe muscle disease that shortens the life expectancy and quality of life for approximately 300,000 boys and young men worldwide. Even though these potential treatments that are currently in the news have not Read more »


    Posted on April 9, 201310:26 pm

    It’s been almost 10 years since CureDuchenne funded a little know biotech company, Prosensa, in the Netherlands.  With only $10,000 in our bank account, we committed to fund a $1.3 Million project to develop antisense (exon skipping) for Duchenne.  A few years later, venture capital firms validated our investment with several million dollars to continue their research.  Read more »

  9. Update from GSK on drisapersen (previously GSK2402968)

    Posted on April 8, 20132:34 pm

    GSK will be announcing results from their Phase llb study this week.  We will keep you updated on the news as it comes in.  Here is the note from GSK: ———————————————————————————————————————————————- To keep you updated on the progress of the drisapersen (previously GSK2402968) clinical development program, as you have requested, I am writing to inform Read more »

  10. Sarepta Therapeutics Announces Eteplirsen Demonstrates Sustained Benefit on Walking Test Through 74 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

    Posted on April 5, 20131:44 pm

    Data to Be Presented at the Muscular Dystrophy Association Scientific Conference CAMBRIDGE, MA–(Marketwired – April 05, 2013) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced updated data from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results at 74 weeks Read more »