New Duchenne Therapies in Development
Mutation-specific approaches like exon skipping are not our only irons in the fire. Many of these other therapeutic approaches will be entering the clinic by the end of this year or the next, providing a variety of options for participating in trials.
At Long Last, A Victory!
This brave and beautiful community has demonstrated in various ways, that this one life will be well-spent, focused on changing the landscape for our children. Today was a day we don’t see nearly enough in our community today was a victory! Learn more about what today's news from Sarepta may mean for your child, as well as what we can expect next.
Sarepta Announces Plans to Submit New Drug Application to FDA for Eteplirsen for the Treatment of Duchenne by Year End 2014
Sarepta Therapeutics today announced it plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) by the end of 2014 for the approval of eteplirsen for the treatment of Duchenne. Eteplirsen is Sarepta’s lead exon-skipping drug candidate in development for the treatment of patients with Duchenne who have a genotype amenable to skipping of exon 51.
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.
Learn more about what Sarepta's announcmeet regarding eteplirsen may mean for your child, as well as what we can expect next.
April 21, 2014 | Read more