19 Jun
  • By cure.duchenne
  • Cause in

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 […]

READ MORE
28 May
  • By cure.duchenne
  • Cause in

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 […]

READ MORE
28 May
  • By cure.duchenne
  • Cause in

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 […]

READ MORE
06 May
  • By cure.duchenne
  • Cause in

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 […]

READ MORE
30 Apr
  • By cure.duchenne
  • Cause in

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 […]

READ MORE
24 Apr
  • By cure.duchenne
  • Cause in

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 […]

READ MORE
23 Apr
  • By cure.duchenne
  • Cause in

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 […]

READ MORE
23 Apr
  • By cure.duchenne
  • Cause in

Eteplirsen Notes from the MDA Conference

This afternoon Sarepta presented data on their trial for their lead candidates to skip exon 51 in Duchenne muscular dystrophy at the MDA Conference in Washington DC.  Most of this has been presented recently, there are a few more weeks of data here. Dr. Jerry Mendell presented the 74 week data for eteplirsen: Eteplirsen continues to […]

READ MORE
22 Apr
  • By cure.duchenne
  • Cause in

DEPARTMENT OF DEFENSE DUCHENNE MUSCULAR DYSTROPHY RESEARCH PROGRAM FISCAL YEAR 2013 STRATEGIC PLAN

The Fiscal Year 2013 (FY13) Defense Appropriations Act provides for $3.2 million (M) to the Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP) to support innovative, high-impact Duchenne muscular dystrophy research. This program is administered by the US Army Medical Research and Materiel Command through the Office of the Congressionally Directed Medical Research Programs […]

READ MORE
15 Apr
  • By cure.duchenne
  • Cause in

Sarepta Therapeutics Announces FDA Will Consider Accelerated Approval for Eteplirsen After Further Review of Data on Dystrophin and Clinical Outcomes

Eteplirsen Manufacturing and Clinical Activities Continue as Planned CAMBRIDGE, MA–(Marketwired – April 15, 2013) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT) today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding a potential application for accelerated approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). The FDA has […]

READ MORE