Our doctors and clinical teams are continually involved in research aimed at determining the causes and most effective treatments for health conditions that impact children. Often, this research involves clinical trials of diagnostic and treatment options that offer great promise, but are not yet widely available.
Dr. Amy Harper and Research Coordinator Kathy O’Hara in our neurology team are involved in clinical trials for pediatric muscular dystrophy treatments. On Friday, CHoR/VCU became the first site to open for recruitment and to screen the first patient in a new clinical trial. This six-year-old and his family have anxiously awaited this trial, and today, he was the first patient in the nation to be screened.
“This specific treatment has the potential to help preserve the heart and muscles in patients with Duchenne muscular dystrophy,” said Amy Harper, MD.
In DMD the body is not able to produce dystrophin, a protein that muscles need to work properly. Without dystrophin, the muscles become weak and damaged over time. There is currently no cure.
“VCU is quickly becoming more recognized as a site for muscular dystrophy clinical research, which reflects the caliber and dedication of our research efforts. This is our fourth treatment trial in two years, with others on the horizon,” added Harper. “We are determined offer all kids with DMD the opportunity to participate in trials like this one. Together, we are muscle strong!”
The team also has trials planned to target other forms of muscular dystrophy and neuromuscular disorders, including myotonic dystrophy, limb-girdle muscular dystrophy and spinal muscular atrophy.
At CHoR, we’re dedicated to making life better for kids by caring for them today and paving the way for an even healthier future.
Could your child benefit from our team approach to muscular dystrophy care? Learn more about our MD clinic or call us at 804-828-CHOR to schedule an appointment with the team.