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 […]

  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 […]

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 […]

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 […]

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 […]

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 […]

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 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 […]

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 […]