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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06 May
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Summary of GSK – Drisapersen Webinar

DRISAPERSEN UPDATE: results from a randomized, double blind, placebo-controlled Phase II clinical trial Webinar with GlaxoSmithKline took place on Monday 6th May 2013, 9:00 am PDT. The webinar was to inform and update the Duchenne community on the Phase II clinical trial results for GSK2402968 (drisapersen) (DMD study 114117). Drisapersen is a 2’-O-methyl-phosphothioate oligo designed […]

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30 Apr
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Best Practices in Clinical Study Design for Rare Diseases

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 […]

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24 Apr
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GSK Update on Extension Study for Disapersen

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 […]

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23 Apr
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GSK Drisapersen Notes from the MDA Conference

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 […]

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