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. […]
CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, announces the 6th Annual Dealing for Duchenne gala will be held on Saturday, May 11 at 6:00 p.m. under the stars at the legendary Sony Pictures Studios in Culver City, Calif. This star-studded event will benefit the Center for Duchenne Muscular Dystrophy at UCLA. Actor Joel Murray (The Artist, God Bless America, Mad Men, Two and a Half Men) will host the Dealing for Duchenne charity poker tournament to raise funds to support transformative research and provide quality care for children living with Duchenne, a progressive muscle-wasting disease that impacts 1 in 3,500 boys. Boys are usually diagnosed by 5, in a wheelchair by 12 and most donÌ¢âÂã¢t survive their mid-20s.
Jim Ruetz of Oak Creek, Wisconsin, will embark on a 4,000-mile, 12-day motorcycle trip across 12 states in the Cajun-Parrot Run to benefit CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy. Ruetz starts his journey on June 5 from Oak Creek. In route, heÌ¢âÂã¢ll stop in New Orleans, his furthest waypoint is Key West, Fla., Ruetz will make his way back to Wisconsin on or near June 18.
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 […]
