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 […]

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 […]

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 […]

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. […]