When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), a group of diseases that causes muscles to weaken and break down over time. The news was overwhelming for her young mother, and it puzzled her doctors, as this rare and progressive genetic disorder primarily affects boys, not girls.
Raniya’s DMD affected everything from walking and breathing to blood circulation. For 11 years, Raniya has depended on medication, physical therapy, and a wheelchair to help manage her worsening symptoms.
First Girl in the World to Receive Elevidys Gene Therapy
But now, a new gene therapy is offering the family hope. Raniya, now 16, is the first girl in the world to receive the groundbreaking gene therapy, thanks to physicians at UHealth – University of Miami Health System and Holtz Children’s Hospital. Additionally, her younger brother, Amir Monstanto, 8 – who also has DMD – received the treatment alongside her. [Read More: University of Miami Health System and Holtz Children’s Hospital Physicians Evaluated the Effectiveness of ELEVIDYS]
“I have been working in neurogenetics for 30 years, and it’s amazing to see the dream of gene therapy being realized,” Dr. Lopez-Alberola said. “It’s a wonderful opportunity to treat these patients and see the impact it’s having. There is much to celebrate.”
