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. […]
Thanks to GSK, Prosensa and Sarepta and, the landscape is changing for the patients and families that are suffering from Duchenne, a severe muscle disease that shortens the life expectancy […]
It’s been almost 10 years since CureDuchenne funded a little know biotech company, Prosensa, in the Netherlands. With only $10,000 in our bank account, we committed to fund a $1.3 Million project […]
GSK will be announcing results from their Phase llb study this week. We will keep you updated on the news as it comes in. Here is the note from GSK: […]
