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
Added by Pat Furlong on October 17, 2013 at 9:00am — No Comments
For many years, patient representatives have been invited to participate in meetings around drug development as the patient voice is extremely important if reviews are to understand how patients weigh benefit and risk within the context of their disease. I was honored to present earlier this week at meetings for NINDS and the FDA. Below are some of the notes I took and some of the information I heard that I wanted to share with you all.
How does it work? FDA issues guidance…Continue
Added by Pat Furlong on September 13, 2013 at 2:21pm — No Comments
Today is a landmark day for the entire Duchenne community. Not too long ago, the Duchenne therapy landscape was a barren field with little to no life. Thanks to a commitment to our patients and the field by the U.S. government, international governments, the venture community and industry, this landscape has changed markedly to the point where today we have the announcement for a…Continue
July 9, 2013.
PPMD met with Dr. Janet Woodcock and leadership from the Office of New Drugs, Medical Policy and the Division of Neurology Products, as well as, staff from the Office of Strategic Programs, Office of Health and Constituent Affairs. The purpose of the meeting was to continue our efforts to provide the agency relevant information and data about Duchenne that will encourage/enable them to be flexible in their review process.
Added by Pat Furlong on July 12, 2013 at 10:39am — No Comments
On July 9, PPMD will be meeting with individuals from FDA to include CDER, Division of Neurology, Office of Strategic Programs and Staff from the Office of health and Constituent Affairs.
The topics for discussion will include a review of PPMD’s Board Policy, developed in 2011. This policy embraces the following principles supporting an enhanced regulatory framework to advance development of drugs for rare diseases:
Added by Pat Furlong on July 3, 2013 at 1:30pm — No Comments
Today, I will be speaking with Treat NMD and European Medicines Agency (EMA) in response to their draft guidance on clinical studies in Duchenne muscular dystrophy. Elizabeth Vroom from Duchenne Parent Project in the Netherlands, Filippo Buccella from Parent Project Onlus in Italy, and I will be speaking on topics like clinical trials, inclusion criteria, and…Continue
June 7/8, 2013
In February, 2013 EMA (Europeans Medicines Agency) released draft “Guidelines on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy” open for comment. This is the result of advances in basic and clinical research with an ever increasing number of clinical trials, recruiting small numbers of patients for these studies. This has raised a number of issues to include study design, the choice of appropriate…Continue
Added by Pat Furlong on May 30, 2013 at 1:26pm — No Comments
Several weeks ago, I was invited to participate in a discussion focused on how large pharmaceutical companies should communicate with advocacy; how communication needs to be different in rare disease; and more specifically what advocates in rare diseases expect, want, and need from pharmaceutical companies. I was one of…Continue
Added by Pat Furlong on May 22, 2013 at 12:30pm — No Comments
Wading through policy is no easy task. But imagine policy as the white lines on the road, the boundaries within which FDA operates. Building our case, showing how Duchenne fits into those white lines and demonstrating how and when Accelerated Approval makes perfect sense is the purpose of PPMD’s white paper. …Continue
Added by Pat Furlong on May 20, 2013 at 11:51am — No Comments