HOUSTON, Aug. 19, 2025 – Texas Children’s has announced that a three-year-old girl has become the first patient successfully treated with the FDA-approved gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency.
AADC deficiency is a rare inherited neurological disorder that prevents the brain from producing dopamine and serotonin, essential for movement, mood and nervous system functions. Only about 350 cases have been reported worldwide, and life expectancy has historically been five to seven years.
According to Texas Children’s, the hospital was the largest U.S. contributor to the clinical trial that led to approval of the therapy, known as Kebilidi (eladocagene exuparvovec-tneq). The drug was approved Nov. 13, 2024, and is administered through a minimally invasive stereotactic neurosurgery in which a neurosurgeon infuses the treatment directly into the brain’s putamen.
Dr. Daniel J. Curry, Director of Functional Neurosurgery and Epilepsy Surgery at Texas Children’s and Professor of Neurological Surgery at Baylor College of Medicine, performed the six-hour procedure. “Before now, AADC deficiency was a hopeless diagnosis. With this treatment, we’ve entered a whole new era where we can deliver solutions to formerly untreatable genetic problems,” Curry said.
The patient was diagnosed at 18 months following genetic testing after showing developmental delays and poor muscle control. After the procedure, she stayed in the hospital for two weeks for monitoring before being discharged. She is expected to show measurable progress within two to six months, but doctors report early signs of improved energy and mood.
Texas Children’s Intracerebral Gene Therapy Program, developed by Curry, is also researching therapies for Rett syndrome, NGLY-1 deficiency, Batten’s disease and other neurodegenerative conditions.
For more information about Texas Children’s and its pediatric neurosurgery program, visit www.texaschildrens.org.a
