Small start, big gains: Loma Linda brings hope to a young patient
Loma Linda University Children’s Health
Loma Linda, Calif.
Photo of Aron and his parents, courtesy of Loma Linda University Children's Health
Late in her pregnancy, Kati Gyulai received unexpected news: Her baby’s limbs were measuring much shorter than average. Her doctors feared it may be a type of skeletal dysplasia, a condition that can be fatal. Unable to verify the diagnosis prior to birth, Kati, her husband, Alpar, and their care team braced themselves for a potentially complicated delivery.
When baby Aron arrived, he was instead diagnosed with achondroplasia — the most common form of dwarfism. From the start, Loma Linda University Children’s Health in California was by their side, offering expert care and guidance. Aron faced some early challenges: sleep apnea, hearing loss and eye alignment issues. But Loma Linda’s team of pediatric specialists coordinated surgeries, therapies and regular check-ins to make sure Aron stayed on track developmentally.
Loma Linda also helped Aron access Voxzogo, a medication that directly targets the underlying genetic cause of the condition.
“Voxzogo works at the growth plates of bones to counteract the overactive FGFR3 signal, allowing bones to grow more normally,” said Hua Wang, M.D., Aron’s geneticist at Loma Linda University Children’s Health. “It represents a major breakthrough. For the first time, we have a treatment that can improve growth and possibly body proportions in children with achondroplasia.”
After Aron participated in two clinical trials led by other providers, Dr. Wang helped Kati and Alpar navigate insurance hurdles so Aron could gain access to Voxzogo once it received approval from the Food and Drug Administration. Within a year of treatment, Aron saw significant limb growth. Now 5 years old, he is doing well and recently transitioned from special education preschool to a general classroom.