End Duchenne eNews: Transforming Duchenne Care
Catch up on the latest research and community updates in this month's End Duchenne eNews.
A New Care Guide to Give to Your Provider – Imperatives for Duchenne Care
Health care providers care for many people, with many diagnoses – far too many for any one provider to be an expert in them all. Make sure that you are receiving the best possible care by giving your provider this NEW "cheat sheet" guide to comprehensive Duchenne care.
Prosensa Update on Drisapersen (webinar)
Prosensa presented a status update on Drisapersen and its follow-on compounds in a patient focused webinar hosted by United Parent Projects Muscular Dystrophy (UPPMD) on Tuesday, March 25th at 1 PM ET.
PPMD Awards $100,000 in Exploratory Grants
PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. PPMD has also awarded ReveraGen BioPharma an award of $50,000 to determine if small snippets of RNA called "microRNAs" can affect the efficiency of exon-skipping.
NEW Certified Duchenne Care Center Program
PPMD is proud to award Nationwide Children’s Hospital as our first Certified Duchenne Care Center. Every person with Duchenne deserves the best care and treatment possible, and has the right to know what clinics meet optimal standards. This program will do just that.
Action Alert: Ensuring Federal Agencies Working on Duchenne Receive Proper Funding
While the MD-CARE Act remains a top priority, we need to also make sure that federal agencies receive adequate funding and direction for the programs that are so critical to our community. One tool we use to ensure that funding and direction is our Congressional funding letter. The investment to date is over $250 million on Duchenne let's keep adding to that number. It takes less than a minute to send your message to Congress.
Registration for PPMD's 20th Annual Connect Conference is now open
This year marks PPMD's 20th Annual Connect Conference in Chicago, June 26-29, 2014. Join families, physicians, researchers, industry leaders, and experts of all kinds as we gather to speak face-to-face about Duchenne.
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.
PPMD continues to demand that the FDA rapidly approves treatments that show safety and effectiveness.
March 7, 2014 | Read more