1. Summary of the DIA and NORD Conference

    Posted on October 18, 201310:57 pm

    The “U.S. Conference on Rare Diseases & Orphan Products: The New Era in Health Care” hosted by DIA and the National Organization for Rare Disorders (NORD) was held in Bethesda, MD from October 7-9. CureDuchenne provides a summary of the conference below.  Thank you Abby Bronson for attending on behalf of CureDuchenne and sharing your Read more »

  2. Highlights from the Sarepta Webinar Regarding Etiplersen and Additional Exon Skipping Programs for Duichenne Muscular Dystrophy.

    Posted on October 17, 201310:37 pm

    This is a very brief summary of some of the key points from this morning’s webinar hosted by Sarepta and moderated by Chris Garabedian, CEO and Ed Kaye, CMO.  CureDuchenne is proud to have been a supporter in the development of etiplersen and we appreciate this company update. The Duchenne community is keen to help Read more »

  3. Recap of GSK and Prosensa October 15 Webinar

    Posted on 1:42 am

    On Tuesday October 15, GSK and Prosensa conducted a conference call for the Duchenne community, especially for members that may not have been at several of the recent meetings where the two companies reviewed data on several of their clinical trials. CureDuchenne provides a recap of the webinar below. GSK reviewed its phase 3 trial Read more »

  4. GSK and Prosensa will hold a joint webinar on Tuesday, October 15, 2013 at 11 a.m. ET and 8 a.m. PT

    Posted on October 14, 20133:23 pm

    Dear Patient Group Representative, GSK and Prosensa will hold a joint webinar on Tuesday 15th October 2013 at 4:00 PM British Summer Time (GMT+0100) for the DMD patient community.   Pre-registration entry for Patient Group Representatives and their members: You will need to register for the conference call using the Pre-registration web site. After registering, you will be Read more »

  5. GSK/Prosensa Duchenne Community

    Posted on October 11, 201311:09 am

    Dear Patient Group Representative, GSK and Prosensa will hold a joint webinar on Tuesday 15th October 2013 at 8:00 am Pacific, 11:00 am Eastern for the DMD patient community.   Pre-registration entry for   Patient Group Representatives and their members: You will need to register for   the conference call using the Pre-registration web site. After registering,   Read more »

  6. Sarepta Therapeutics to Host Webcast and Conference Call for Duchenne Muscular Dystrophy Community on October 17

    Posted on October 10, 20138:53 pm

    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 Read more »

  7. GSK and Prosensa Will Hold a Joint Webinar on October 15

    Posted on October 7, 20136:09 pm

      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 Read more »

  8. Notes From World Muscle Society 2013

    Posted on 3:17 am

    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 Read more »


    Posted on October 5, 201310:41 pm

    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 Read more »


    Posted on October 4, 201310:18 pm

    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 Read more »

  11. Prosensa to Host Conference Call to Discuss Recent Drisapersen Data Presentations

    Posted on 12:52 am

    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 Read more »

  12. Sarepta Therapeutics Introduces Online Resource Center on Exon Skipping for the Duchenne Muscular Dystrophy Community

    Posted on October 2, 20133:29 pm

    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, Read more »