Two weeks ago, I was invited to present the story of Duchenne muscular dystrophy to Acceleron’s partner, Shire Pharmaceuticals. I walked in the door to find myself surrounded by the word “BRAVE.”  Brave is Shire’s approach, describes their commitment to rare disorders and their view of individuals with rare disorders.  “Brave” was on desks, next to photographs of patients, “Brave” was on walls, with descriptions of rare conditions.  “Brave” was the character of the people on Shire’s team.…

The third panel for the One Voice Summit, moderated by Debra Lapin, focused specifically on the accomplishments and possible gaps in the MDCC Action Plan.  Members of industry and researchers made up the panel.  Jasbir Seehra and Chris Garabedian represented industry while Se-Jin Lee, and Eric Hoffman represented research.  Bob McDonald is a parent PPMD Board member who also was on this panel.  

The discussion began with Eric Hoffman’s review…

The second panel for the One Voice Summit, moderated by David Zook, focused on Quality of Care and Quality of Life issues.  Kathryn Wagner, Mark Swanson, Katie Bushby, and Craig McDonald were panelist joined by a committee with Jerry Mendel, Katherine Mathews and Jen Garofalo.  

The panel took turns addressing aspects of the MDCC Action Plan addressing programs such as the MD STARnet, comparing and contrasting the Plan to efforts undertaken…

Pat Furlong opened the One Voice Summit for Duchenne Muscular Dystrophy welcoming attendees.  She provided background of the MD CARE Act and the accomplishments over the past ten years including the Muscular Dystrophy Coordinating Committee’s Action Plan for muscular dystrophy.  Pat spoke about those who helped initiate Parent Project Muscular Dystrophy’s entry into the advocacy including meetings and advice provided related to Senators Arlen Spector and Roger Wicker, Joel Wood…