Time and again in our Duchenne community, we see brave kids and their warrior parents work to change the landscape. After all, it’s that exact spirit upon which PPMD was founded. And with that same spirit that family after family has transformed their personal moments into broader movements that have benefited us all.
On May 18, the FDA Pediatric Advisory Committee convened to determine whether to allow for the protocol of Sarepta’s ESSENCE trial to…
ContinueAdded by Annie Kennedy on May 26, 2017 at 2:45pm — No Comments
Today’s Pediatric Advisory Committee Meeting resulted in a unanimous decision from committee members to recommend the use of in-dwelling ports in Sarepta's ESSENCE clinical trial. This is an important step forward for our community,…
ContinueAdded by Annie Kennedy on May 18, 2017 at 4:30pm — No Comments
As our clinical trial pipeline unfolds and therapies become available, navigating the landscape is becoming increasingly complex. But today our landscape shone a bit brighter as the sun rose on the FDA campus early this morning and we watched members of our Duchenne community arrive.
Because today – our Duchenne…
Added by Annie Kennedy on May 18, 2017 at 10:00am — No Comments
PPMD's SVP of Legislation & Public Policy, Annie Kennedy and Michelle Puryear, MD, PhD, testified at the Advisory Committee on Heritable Disorders in Newborns and Children last week.
On the heels of a recent publication in the International Journal of Neonatal Screening, Dr. Puryear provided an update of the therapeutic pipeline and some of PPMD's activities around developing infrastructure to support a pilot for newborn screening for…
ContinueAdded by Annie Kennedy on May 16, 2017 at 4:40pm — No Comments
PPMD is proud to join dozens of other rare disease nonprofit organizations in asking Congressional leaders to reauthorize the FDA user fee agreements.
The current FDA user fee agreements are the culmination of months of negotiation between FDA and the medical product industry, with significant input from the patient advocacy community.
ContinueAdded by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments
Earlier this month, PPMD was proud to be among seven patient advocacy organizations who took a lead in laying out a joint set of principles to guide any efforts that seek to change the process of accessing unapproved therapies outside of a clinical trial, also known as compassionate use or expanded access.
The patient advocacy organizations include:
Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments
PPMD is thrilled to announce that on May 4 the BENEFIT Act was introduced in the Senate by longtime Duchenne community champions, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN).
Over the past five years, Congress has made considerable progress in driving forward policies and procedures to ensure the patient perspective is considered by Food and Drug Administration (FDA) reviewers evaluating candidate drugs and other medical products. As a result of…
ContinueAdded by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments
We may have lost the battle — but the war is far from over.
Some days are hard to make sense of. Today feels like one of those for many as we watched the House votes tally this afternoon.
This community knows better than most that our healthcare system needs repair and innovation. But the proposals that have been brought forward have not represented enhancements to healthcare…
Added by Annie Kennedy on May 4, 2017 at 6:00pm — No Comments
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