» Drisapersen update from Prosensa Action Duchenne

» Drisapersen update from Prosensa Action Duchenne: "Prosensa issued an update today to the parent-led organisations on their lead exon skipping compound to skip exon 51, drisapersen; following their recent UPPMD webinar on the 25th of March.
Whilst Prosensa are finalising the transfer from GSK, they also continue to embark on their program to re-dosing the first cohort of boys who participated in the trial in the third quarter of this year and also communicating their plans and regulatory paths forward forward, by the end of June."



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Webinar: Sarepta Therapeutics - PPMD Community

Webinar: Sarepta Therapeutics - PPMD Community: "Sarepta Therapeutics, Inc. will host a webcast and conference call for the Duchenne muscular dystrophy (DMD) community on Tuesday, May 13, 2014, beginning at 11:00 a.m. EDT (8:00 a.m. PDT / 5:00 p.m. CEST).

Chris Garabedian, president and chief executive officer, and Edward Kaye, M.D., chief medical officer, will provide information and answer questions about Sarepta's DMD development program, including upcoming clinical trials. Sarepta has invited DMD community representatives from leading patient advocacy groups to participate in the question and answer portion of the webcast."



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At Long Last, A Victory! - PPMD Community

At Long Last, A Victory! - PPMD Community: "This morning, Sarepta announced that its discussions with the Food and Drug Administration (FDA) over the last several months have borne fruit—the FDA has agreed to allow Sarepta to submit a New Drug Application (NDA) for eteplirsen.  The company plans to submit the application by the end of this year and should have a decision from the FDA by mid-2015 about whether or not it will be approved.   Over the last several years, the chances that a drug will go on to be approved after an NDA is allowed is well over 50%. "



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Sarepta Therapeutics to Submit New Drug Application to FDA for Eteplirsen by the end of 2014 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Sarepta Therapeutics to Submit New Drug Application to FDA for Eteplirsen by the end of 2014 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Sarepta Therapeutics announced yesterday their plans to submit a new drug application (NDA) for eteplirsen to the US Food and Drug Administration (FDA) by the end of this year. Eteplirsen is Sarepta's lead candidate to skip exon 51."



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Clinical trial recruitment updates - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Clinical trial recruitment updates - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Action Duchenne are in continuous dialogue with healthcare professionals, leading biotech and pharmaceutical companies regarding their growing Duchenne pipeline. A snapshot of clinical trial recruitment updates are below.

FOR-DMD (Finding the Optimum Regimen of Corticosteroids for DMD)

This compares daily prednisone and deflazacort regimes against one of the more commonly used intermittent regimes in boys living with Duchenne. "



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Certified Duchenne Care Center Program - PPMD Community

Certified Duchenne Care Center Program - PPMD Community: "Right now, there are more than 10-12,000 young people diagnosed and living in the United States with Duchenne. These young people are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Families want care and treatment to be transparent; they want to know who is delivering care to their child, what comprehensive care looks like, and which centers are delivering that care. For this reason, a group of experts agreed to give families the information that they deserve and to develop a methodology and process by which to certify clinics delivering comprehensive care, and then measure results.

PPMD’s Transforming Duchenne Care Initiative (TDCI) set out to reduce discrepancies in care. The goal – to make comprehensive care, aligned with the recommendations of the DMD Care Considerations Working Group (DMDWG, which published the CDC Care Considerations standards of Duchenne care, made available in 2009 and published in 2010), available and accessible to as many patients across the US as possible."



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Webinar: Prosensa Update on Drisapersen - PPMD Community

Webinar: Prosensa Update on Drisapersen - PPMD Community: "Prosensa presented a status update on Drisapersen and its follow-on compounds in a patient focused webinar hosted by United Parent Projects Muscular Dystrophy (UPPMD) on Tuesday, March 25th at 1 PM ET.

The webinar was presented by Giles Campion MD, Chief Medical Officer and Senior Vice-­‐President R&D of Prosensa and featured the following guest speakers:
Craig Campbell MD MSc FRCPC, Head of Section of Pediatric Neurology at London Health Sciences Centre, Associate Professor at Shulich School of Medicine of Western University, Associate Scientist at Child Health Research Institute
Craig M. McDonald MD, Professor and Chair of Physical Medicine & Rehabilitation, Professor of Pediatrics at University of California Davis School of Medicine"



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