Wednesday, 30 April 2014

» 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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Wednesday, 23 April 2014

Wednesday, 16 April 2014

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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