Each month for the last couple of years we have featured people with Duchenne who have made an impact on their community and our community in some significant way. Whether working with their parents to host a fundraising event, participating in a race, speaking in Washington, D.C., or sharing their story…Continue
Added by Will Nolan on December 16, 2013 at 2:30pm — No Comments
At yesterday’s Duchenne Policy Forum, Vice President of Research Sharon Hesterlee, PhD, tweeted throughout the day, reporting to the community what transpired during this historic meeting. We have compiled these tweets and fit them into the…Continue
12/12/13 8:30 AM
There are days when the stars align just right. Seems to me, December 12, 2013 was one of those days.
Eighteen members of the FDA arrived and were seated around a U-shaped table near the front of the…Continue
Why the December FDA Policy Forum is important to every Duchenne family – from rare mutations to Exon 51
Guest post Lance Hester. Lance is a PPMD Board Member and lives in Gig Harbor, Washington, with his wife, Janelle, and their two boys, Brayden and Micah. Micah was diagnosed with Duchenne in 2005.…Continue
Added by PPMD on December 9, 2013 at 1:00pm — No Comments
Due to overwhelming interest in PPMD’s Duchenne Policy Forum on December 12 in Silver Springs, MD, registration is now closed. We are at capacity and unfortunately there will be no onsite…Continue
Added by Ryan Fischer on December 6, 2013 at 11:30am — No Comments
For our families, Duchenne research can’t move fast enough.
There is no lack of ideas or interest—7 public companies and more than 15 biotechs are focused on finding treatments for Duchenne. But promise is only one part of the story. We need to actually change the landscape and bring drugs to market faster.
You can help make it happen with one simple action:…Continue
Added by Pat Furlong on December 4, 2013 at 11:00am — No Comments
PPMD’s research strategy has long had three distinct facets—to develop treatments Better, Faster, and Now. In the past, I have talked about how we have teamed up with TREAT-NMD to use its panel of experts on Duchenne and drug development to review projects with the goal of putting…Continue
2013 has been a year of incredibly high highs and incredibly low lows. Sometimes those lows have overshadowed all the highs—and for good reason. It’s impossible to put into words how devastating the setbacks are.
But our community is and has always been stronger than the setbacks. We may be fighting a disease that takes away muscle, but you are the strongest people I’ve ever known. It’s during our biggest challenges that strength matters most, and I know we’ll get through this…
Day after day, we hear the heartbreak of Duchenne: a 7 year-old boy with the new discovery of decreased function and significant fibrosis, a 9 year-old boy with more than a 10% decrease in function from just 1 year ago, an 11 year-old boy dying from heart failure.
Even if you are…Continue
Added by Kathi Kinnett on November 22, 2013 at 2:04pm — No Comments
In a few weeks, PPMD will be hosting a Policy Forum on clinical trials of experiment agents This is a landmark meeting that will include participation from the FDA. It will also include Ansel Lurio, our Featured…Continue
Added by Will Nolan on November 15, 2013 at 10:00am — No Comments
Yesterday was a disappointing day for all parents, grandparents, aunts, uncles, and others touched by Duchenne. Like most of our community, I awoke to the news that multiple factors have led Sarepta to announce that it would delay the filing of its much-anticipated…Continue
By now many of you may have heard the news that the FDA has called the new drug application for the exon 51 skipping drug eteplirsen premature. They have requested instead that a larger phase III study be conducted before they will consider the application for approval. PPMD, which provided travel support to families who…Continue
The Child Neurology Society Annual Meeting, held in Austin 10/30-11/2, was a busy meeting, packed with interesting sessions and attendees from all of the world. While there was not a lot of late breaking news, it was an excellent opportunity for providers engaging in both clinical…Continue
Added by Kathi Kinnett on November 7, 2013 at 10:30am — No Comments
PPMD is proud to host a Policy Forum on clinical trials of experiment agents on December 12, 2013 from 8.30AM until 4.30PM in Silver Springs, MD. We are especially pleased that key Duchenne-community…Continue
Added by Pat Furlong on November 6, 2013 at 3:00pm — No Comments
When parents of children with Duchenne enroll their children in a clinical trial there is often a sense of participating in something bigger than themselves—of course you hope that your child may benefit from a new therapeutic approach, but you also know that all those days of missed school,…Continue
Added by Sharon Hesterlee on October 29, 2013 at 11:00am — No Comments
When Chris and Patrick were diagnosed, there was no genetic testing. No therapies were targeted to the “CODE” and no way to DECODE Duchenne. We made assumptions about carrier status and lived many years with only assumptions. I thought my mother was a carrier. I knew I was a carrier. The doctor made that…Continue
Added by Pat Furlong on October 23, 2013 at 10:30am — No Comments
Added by Pat Furlong on October 17, 2013 at 9:00am — No Comments
When a family hosts a fundraising event for Duchenne, we are thrilled and extremely grateful. When a family hosts the same event six years in a row, we are overwhelmed! This year the Pritchard family, along with their friends – Meghan and Jason Alpern – in Matthews, North Carolina will host the…Continue
Added by Will Nolan on October 15, 2013 at 11:30am — No Comments
We all know it: rare diseases, especially progressive pediatric disorders like Duchenne, are different than common diseases. The FDA knows it too, but they don’t have a plan for how to treat them differently. They are working on different ways to weigh the benefits and risks of treatments for…Continue
Added by Holly Peay on October 10, 2013 at 9:30am — No Comments
I was privileged to share some of the data PPMD has collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences, and were quite interested in how they might…Continue
Added by Holly Peay on October 7, 2013 at 10:58am — No Comments