Contemporary Cardiac Issues in Duchenne
Heart issues don't just affect some people with Duchenne; they affect ALL people with Duchenne and heart failure remains one of the leading causes of death in Duchenne. Cardiac concerns continue to be a top priority for PPMD. One way we have taken action to further explore these heart issues is by joining together with the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH) to develop a cardiac working group.
Nutrition, Energy, and Duchenne
During her years of clinical practice, Kathi Kinnett, Vice President of Clinical Care was often asked, “Is there something that I can feed my child to help slow down, or stop, the effects of Duchenne?” The absolute answer is we don’t know. But a new article out this week has endeavored to address this question.
Take Action Now!
ONE minute of your time will mean a lifetime for those with Duchenne. As members of the Duchenne community are on Capitol Hill meeting with Members of Congress, we need you to take just ONE minute and take a simple action that will let your Member know how critical their support of this legislation is.
DuchenneConnect Receives $970,000 Award
DuchenneConnect will be one of 29 members of PCORnet, the National Patient-Centered Clinical Research Network. The goal of PCORnet is to create a large, highly representative, national network for conducting clinical research. We hope to ask and answer important questions about Duchenne and Becker using the huge, connected network.
End Duchenne eNews: Raising Our Voices
Catch up on the latest research and community updates in this month's End Duchenne eNews.
Webinar: How You Can Help PPMD’s Advocacy Agenda
On February 11th, Pat Furlong, Ryan Fischer and Dave Zook (FaegreBD) provided an overview of PPMD’s overarching advocacy strategy, laying out our goals for 2014 and discussing how you can get involved at our Advocacy Conference in Washington, DC and/or advocate from your home.
Webinar: ACT DMD Update with PTC
On February 5, 2014, Dr. Jay Barth, Vice President for Clinical Development at PTC Therapeutics, presented an update about PTC’s Phase 3 study of ataluren, now known as the Ataluren Confirmatory Trial in DMD (ACT DMD).
Register for the 2014 Advocacy Conference (Feb 23-25)
You are invited to join us in Washington (February 23-25) for what is going to be another critical year in Duchenne advocacy. As you know our efforts last year were focused on collecting cosponsors for the MD-CARE Act (MDCA) reauthorization. To date we stand at 77 cosponsors in the House and 17 in the Senate.
FDA Guidance: Representing the Duchenne Community
On Monday, February 24, members of the Steering Committee and working groups will provide progress to date and may request additional comments and opinion about particular topics. This will take place in conjunction with our Annual Advocacy Conference.
PPMD Funds Kinnect Project
Recognizing the need to enhance trial participation for those who are non ambulatory, PPMD has invested in the development of endpoints to measure upper body function. One project PPMD is funding uses the Microsoft Kinect system to measure the "functional space" that a person can reach.
PPMD continues to demand that the FDA rapidly approves treatments that show safety and effectiveness.
March 7, 2014 | Read more