Amy Harper, MD ’98 Shares Insight on the Rarity of Muscular Dystrophy
Congratulations to American University of the Caribbean School of Medicine (AUC) alumna Amy Harper, MD '98 who was recently promoted to professor, Department of Neurology at Virginia Commonwealth University (VCU) Health in Richmond, VA! Dr. Harper is also the director of pediatric neuromuscular clinics at Children’s Hospital of Richmond at VCU.
With a primary interest in muscular dystrophy research, Dr. Harper has conducted numerous research studies and clinical trials. She currently serves as the primary investigator in 15 studies. Her interest in muscular dystrophy started during her fellowship in neurodevelopmental disabilities at Oregon Health and Sciences University. Through this fellowship, Dr. Harper earned her certification in child neurology and became eligible for the neurodevelopmental disabilities board.
In 2016, Dr. Harper brought the first treatment trial to VCU Health for Duchenne Muscular Dystrophy (DMD), which eventually expanded to a translational center that conducts research on several other types of muscular dystrophy including Limb-girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy, Congenital Muscular Dystrophy and Myopathy Spinal Muscular Atrophy.
“In general, muscular dystrophies and most pediatrics muscular diseases are rare diseases. It wasn’t until the 1980s that we really knew the gene that caused DMD. Fast forward to 2021 and we now have at least three FDA approved gene-modifying therapies,” says Dr. Harper. She and her partners now conduct investigator studies with National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), and Muscular Dystrophy Association (MDA); several of which have led to FDA-approved medications to treat muscular dystrophy.
As director of pediatric neuromuscular clinics, Dr. Harper says that awareness of muscular dystrophy is crucial, as early detection can improve quality of life for patients.
“When applied early, these ground-breaking treatments have resulted in children living relatively normal lives from a disease that could otherwise result in death by age two,” says Dr. Harper. “Of course, this is really time-sensitive, so we have to treat these babies as early as possible, which is why several states are looking to add muscular dystrophy to newborn screenings.”
Dr. Harper hopes to continue expanding muscular dystrophy treatment trials and offering life-saving opportunities to more families. “Each child should have an opportunity to participate in research, so that’s always our goal with the clinic— to offer every child, regardless of age or disease, the option to participate in research.”