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…Continue
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…Continue
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…Continue