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 […]
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 […]
  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 […]
  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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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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