Jackson Health System's 2026 Report to the Community.
Holtz patient is the first female to receive groundbreaking gene therapy for Muscular Dystrophy Diagnosed at age two with Duchenne muscular dystrophy (DMD), Raniya Scott has spent much of her life facing the progressive challenges of the disease. Now 16, she made history as the first female in the world to receive a promising new gene therapy for DMD.
Her doctors at Holtz Children's Hospital are hopeful that the treatment, the first gene therapy approved for Duchenne muscular dystrophy, could help slow the progression of the disease.
Raniya's doctors say she already appears stronger. Read her story.
Genetic testing is transforming infant care at UHealth Jackson Children's Rare Disease Center
Pankaj Agrawal, MD, chief of neonatology at Holtz Children's Hospital, part of UHealth Jackson Children's, is advancing the use of genetic testing to better understand and treat rare diseases in newborns. By analyzing a child's genetic data, he and his team can identify the underlying cause of complex conditions and determine the most effective course of care.
Through whole genome sequencing and other emerging technologies, Dr. Agrawal's team can anticipate how a child with certain genetic markers may respond to medications or treatments. In some cases, they can even develop care plans before symptoms appear. For babies with rare conditions, this kind of early insight can be lifesaving.
Genetic testing is revolutionizing infant health. Learn more.