PPMD’s program, established in 2014, recognizes facilities with outstanding neuromuscular programs providing comprehensive care with recognized standards to all living with Duchenne muscular dystrophy (DMD), a multi-systemic genetic disorder characterized by progressive muscle loss.
CHoR is part of Virginia Commonwealth University (VCU), and its Muscular Dystrophy Association Care Center offers a robust clinical and investigative program that features comprehensive Duchenne care as well as multiple clinical studies on DMD progression and management, a press release states. Under the guidance of Amy Harper, the center treats more than 60 Duchenne patients, promotes research, and recently added two pediatric neuromuscular specialists.
“Children, parents and families are at the heart of what we do every day,” said Harper, MD, a Child Neurology Society member and neuromuscular disease researcher who joined CHoR in 2016. “We’re honored to be recognized by PPMD, a group of dedicated parents and families, for our advocacy and innovation in the field of neuromuscular diseases.”
Kathi Kinnett, PPMD senior vice president of clinical care and CDCC program director, said Harper and her team have, in a relatively short time, made a marked impact.
“CHoR has close collaboration with the other PPMD Certified Duchenne Care Centers in the area,” Kinnett said. “Working together, these CDCCs are able to offer Duchenne patients and families access to most of the currently ongoing clinical trials. We are incredibly excited to add the expertise and compassion of Dr. Harper’s team to our growing network of certified centers.”
The certification program helps ensure that centers maintain the highest standards in clinical and sub-specialty services, including prompt adaptation of new evidence-based knowledge, high-quality research outcomes, and compliance with updated Duchenne Care Guidelines established by the Centers for Disease Control and Prevention.
“By standardizing care at PPMD’s Certified Duchenne Care Centers, many of which are major clinical sites, we are also improving Duchenne research and clinical trials by decreasing variability in care, and increasing the quality of clinical trial outcome measures,” the CDCC states on a webpage. “This results in speeding the time it takes therapies to reach the people who need them.”
The CDCC program is monitored by the 11-member CDCC Certification Committee, which reviews applications and site-visit summaries, and makes recommendations regarding certification. Kinnett said the PPMD gets CDCC inquiries weekly from centers around the world.
“Families in our community turn to PPMD to help identify the centers in the country providing the best in comprehensive Duchenne care and services,” she said. “Clinics and clinicians are realizing the value of the Certified Duchenne Care Center Program and therefore strive to be the best facility they can be for Duchenne patients and families.”
As global recognition of the importance of uniform care standards and guidelines spreads, PPMD expects to soon expand the CDCC program internationally.
Through research funding and patient advocacy, PPMD aims to end Duchenne, a disorder found in about 1 in 3,500 male births worldwide.