05 Sep
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Thank you Fergie for helping Cure Duchenne!

Yesterday, Fergie – a Grammy award-winning singer/songwriter, and new mom! –  joined the ranks of talented and respected celebrities we love.  Over the last few years, Clay Matthews, Ryan Getzlaf, Mack Brown and Scott Niedermayer have spoken out publicly in support of CureDuchenne.  We are very touched by Fergie’s support. Fergie is appearing in a […]

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30 Aug
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Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

Aug 30, 2013   Skipping multiple exons at the same time, by using a combination of antisense oligonucleotides, offers the potential to treat a significant number of Duchenne patients. This would address one of the major limitations of current antisense therapy, in that the approach is “personalized” and designed to skip a single exon. Specifically, […]

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30 Aug
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DMD transcript imbalance determines dystrophin levels

CureDuchenne’s chief scientific advisor, Dr. Mike Kelly reviewed the recent paper by Annemieke Aartsma-Rus and other researchers.   This is complicated but very important science, we attempt here to explain the findings. http://www.fasebj.org/content/early/2013/08/22/fj.13-232025.abstract The most advanced drugs in clinical development for the treatment of Duchenne (the exon-skipping drug candidates’ drisapersen and eteplirsen as well as the stop-codon read […]

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14 Aug
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Prosensa Provides Updates on Duchenne Development Programs

On August 13, 2013, Prosensa’s CEO, Hans Schikan and, Chief Medical Officer Dr. Giles Campion,  provided updates on the Company’s development programs for the treatment of Duchenne.  CureDuchenne is pleased to see the progress that Prosensa has made, now with six exon skipping programs at different stages of development. CureDuchenne was the lead organization that […]

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08 Aug
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Sarepta and Prosensa Announcements This Week

There continues to be good news for the Duchenne community.  We will continue to monitor Duchenne research news and share it with you as it becomes available. Here are three announcements from this week: Sarepta Therapeutics Announces Publication of Eteplirsen Clinical Study Results in the Annals of Neurology Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer […]

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24 Jul
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Cure Duchenne is Pleased that Sarepta Therapeutics Plans to Submit NDA for Eteplirsen in First Half of 2014

  Sarepta Therapeutics announced today plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2014 for the approval of eteplirsen, an exon skipping compound under development for the treatment of Duchenne muscular dystrophy. CureDuchenne, a leader in raising awareness and funding research to […]

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27 Jun
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GlaxoSmithKline’s Drug Drisapersen Receives Breakthrough Status

  CureDuchenne is pleased that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to drisapersen, an exon-51 skipping compound for the potential treatment of patients with Duchenne muscular dystrophy. Drisapersen is being developed by GlaxoSmithKline (GSK) and licensed from Prosensa Therapeutics. CureDuchenne has worked closely with the FDA to expedite Duchenne drugs […]

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19 Jun
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Sarepta Therapeutics Announces Eteplirsen Demonstrates a Continued Benefit on Walking Test Through 84 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

Data to Be Presented Today at the Wells Fargo Securities 2013 Healthcare Conference CAMBRIDGE, MA–(Marketwired – June 19, 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 […]

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28 May
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PROSENSA HOLDING B.V. FILES REGISTRATION STATEMENT FOR PROPOSED INITIAL PUBLIC OFFERING

Leiden, the Netherlands—May 28, 2013—Prosensa Holding B.V., today announced that it has filed a registration statement on Friday May 24, 2013 on Form F-1 with the U.S. Securities and Exchange Commission (“SEC”) relating to the proposed initial public offering of its ordinary shares. The number of shares to be offered and the price range for […]

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28 May
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New Developments with Prosensa and Sarepta for Exon Skipping in Duchenne

On Monday, the Today Show ran a segment about Jenn McNary and her two sons that have Duchenne, a deadly muscle disease that attacks primarily young boys.  Jenn had been passionate and effective in communicating the urgency of getting drugs for Duchenne approved by the FDA.   Jenn’s older son and thousands of boys like him need to […]

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