Tomorrow, November 24, 2015, is a historic day for the Duchenne community as we enter into our first Advisory Committee Meeting with the FDA. While some of you may be attending in person or even participating, we know that many of you are curious as to how the process will unfold…Continue
Added by PPMD on November 23, 2015 at 2:00pm — No Comments
Dr. Stu Peltz from PTC Therapeutics shares his gratitude to the Duchenne community for the contribution families have made to the development of their therapy, Translarna.
Read Dr. Peltz's…Continue
Added by PPMD on November 18, 2015 at 3:00pm — No Comments
For those of us who’ve been in this community for decades, at some point we began to believe that we’d never see the day when products that had been designed specifically with the intent to treat individuals with Duchenne would be…Continue
Added by Annie Kennedy on November 18, 2015 at 11:00am — No Comments
By now, you probably know what to expect from the upcoming Advisory Committee meetings in terms of logistics: how to submit written testimony, how to apply for an open public hearing slot, order of the day, etc. (…Continue
Added by PPMD on November 17, 2015 at 1:00pm — No Comments
For over a year, PPMD has been working with congressional champions and industry partners to help lead an effort to streamlining the regulatory pathways and review processes for targeted therapeutics (which would include such things as 'follow on exons') by clarifying the FDA’s existing…Continue
Many of you have seen – and signed – a petition that circulated recently about preventing Medicare from applying competitive bidding prices to complex technology; an action that will likely reduce access to wheelchair features many of our family members depend on. This effort has now evolved into a legislative effort and we urge all members of our…Continue
Added by Annie Kennedy on November 3, 2015 at 10:00am — No Comments
Last week I participated in a briefing for Congressional staff in honor of the 10th anniversary of the founding of the Critical Path Institute, our partner in the…Continue
PPMD is pleased to announce that late yesterday we submitted the results of our most recent patient-centered benefit-risk assessment (PCBR) study to the FDA. The assessment was done with research partners at the Johns Hopkins Bloomberg School…Continue
Added by PPMD on October 22, 2015 at 7:48am — No Comments
Why Are Written Comments Important? How Are They Utilized?
Written comments are compiled and shared with the FDA Advisory Committee members prior to the date of a product’s/ company’s Advisory Committee meeting. Thus, written comments are a…Continue
Added by PPMD on October 20, 2015 at 3:30pm — No Comments
PPMD is proud to help author and share the attached letter to Dr. Janet Woodcock, Director of the…Continue
Added by PPMD on October 19, 2015 at 11:00am — No Comments
The first annual meeting of PCORI was held in Washington, DC on October 6-8. 1,100 researchers, patients, caregivers, health system and industry representatives, and others were present to hear updates on patient-centered comparative clinical effectiveness research. PCORnet is a “network of…Continue
Added by John D. Porter, PhD on October 16, 2015 at 1:07pm — No Comments
It’s fall, so it must be time for my annual mantra – Get Your Flu Vaccine!!
Everyone, 6 months of age and older, needs to be vaccinated against influenza, and protecting children with neuromuscular disease is especially important. A…Continue
Added by Kathi Kinnett on October 16, 2015 at 1:00pm — No Comments
Added by John D. Porter, PhD on October 16, 2015 at 11:30am — No Comments
After yesterday’s news regarding the Ad Comms for both BioMarin and Sarepta, the community continues to ask questions. PPMD has been working around the clock to get some answers…Continue
Added by Pat Furlong on October 15, 2015 at 3:30pm — No Comments
Today we learned that the FDA will host an Advisory Committee (Ad Comm) meeting on November 24 to discuss the…Continue
Added by Pat Furlong on October 14, 2015 at 4:30pm — No Comments
What’s a PRO?
Several patient-report outcomes (PROs) have been validated for use in people with Duchenne and their caregivers. Patient-report outcomes are a way for patients and families to provide structured input about their own…Continue
Added by PPMD on October 13, 2015 at 11:30am — No Comments
Yesterday, I had the distinct privilege of representing PPMD and our Duchenne community as I sat before a full room of Senate staffers and congressional aides in the Dirksen Senate office building. Our goal was to urge them to press forward on the Innovation for Healthier Americans bill (also referred to as the ‘Innovation…Continue
In the last few weeks we’ve heard the news from several companies working to develop therapies for Duchenne that they have received Rare Pediatric Disease Designations from the FDA. Santhera, Sarepta, Catabasis, and Biomarin.
We as a community have celebrated this news. But…Continue
Added by Annie Kennedy on October 7, 2015 at 4:30pm — No Comments
This is our final update from the 2015 World Muscle Society meeting in Brighton, United Kingdom (click here for updates from previous days). Before providing summaries of some presentations on the…Continue
Added by PPMD on October 6, 2015 at 10:30am — No Comments
Added by PPMD on October 5, 2015 at 10:23am — No Comments