Duchenne Community Knows No Bounds
Today – September 18, 2014, our Duchenne community once again made history by demonstrating that nothing could stop us from ensuring that the life-saving research momentum of our landmark bill would be expanded to reflect the current needs of our…Continue
As you know, PPMD recently facilitated a multi-stakeholder process involving patients, parents, investigators, industry, and other interested parties which culminated in a draft guidance document…Continue
Added by Pat Furlong on August 18, 2014 at 1:00pm — No Comments
Every year I try to escape the real world for a couple of days and retreat with my family to probably my favorite place on Earth – Dowagiac. I’ve reminisced before, but this place is magical to me. It is my childhood, it is summer, it is magic. With so much going on in our community –…Continue
I had no words for Scott’s death. In fact, he had been actively dying for several years. I had hoped that he might see Rebekah for one last time, though I realized they were both too sick and living in different countries made it impossible. The good news is that many people with Duchenne never find…Continue
Added by Pat Furlong on August 4, 2014 at 10:30am — No Comments
The House of Representatives today approved the landmark MD-CARE Act so the law can continue getting results for all patients impacted by Duchenne and other forms of muscular dystrophy. The House approved the bill, H.R. 594, sponsored by Congressman Dr. Michael Burgess of Texas and Eliot Engel of New…Continue
Added by Pat Furlong on July 28, 2014 at 6:12pm — No Comments
If you are always trying to be normal, you will never know how amazing you can be.
― Maya Angelou
What do you want to be when you grow up? The question Dr. Sue Apkon asks every boy with Duchenne (girls too). The question, that for so many years brought tears to our eyes and a lump in our throat. The question that always resulted in mental math... if this happens and that happens, then maybe…. But now, it is reality. Our sons…
Added by Pat Furlong on July 2, 2014 at 11:00am — No Comments
John Porter, PhD is a Program Director at the NIH/NINDS, responsible for managing a portfolio of research grants across neuromuscular disorders. That is his formal introduction, but the other side is that he has long been a friend to me and to our Duchenne community. In January, he announced his intention to…Continue
Added by Pat Furlong on June 28, 2014 at 5:43pm — No Comments
June, 25, 2014 – Today is a landmark day for PPMD and the entire Duchenne community. We are so pleased that at our organization’s 20 year anniversary – after 2 decades of hard work and resolute persistence within the policy, legislative and regulatory arena – we are today able to provide to the US Food and Drug…Continue
Added by Pat Furlong on June 25, 2014 at 11:00am — No Comments
This week the House Appropriations Committee approved the Fiscal Year 2015 Department of Agriculture and FDA…Continue
Added by Pat Furlong on May 30, 2014 at 11:22am — No Comments
EMA grants Conditional Approval for Ataluren
Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional…Continue
One Life on this earth is all that we get, whether it is enough or not enough, and the obvious conclusion would seem to be that at the very least we are fools if we do not live it as fully and bravely and beautifully as we can.…Continue
As a mother who lost two sons to this dreadful disease, I am driven to find every way possible to help bring new therapies to families, to end Duchenne. PPMD supports and agrees with all parents urging the agency to accept Sarepta’s submission for the approval of eteplirsen, as well as any other therapy that shows similar promise. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.
Added by Pat Furlong on March 7, 2014 at 9:30am — No Comments
Sarepta’s 120 week stability data on both the six minute walk test, as well as, pulmonary function is good news for the Duchenne community. For the 12 young men in the study, we are thrilled. And for those waiting, we see hope on the horizon. We congratulate Sarepta for their continued commitment to people with Duchenne and we hope the FDA…Continue
Added by Pat Furlong on February 6, 2014 at 1:30pm — No Comments
Patrick was 8 years old. Steroids were not recommended. There was no imaging and no way to predict his loss of ambulation. Sure, he fell occasionally. Some days more than others. There was one day in the summer of 1988. We had been active most of the day. We went swimming, to the mall, and ended the day with a cookout. Patrick walked and walked. He asked me to help him to the bathroom. He fell. It was one of those FALLS, as if his legs were pulled out from under him. I had no excuses in my…Continue
Added by Pat Furlong on January 23, 2014 at 9:00am — No Comments
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
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
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…
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
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 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