Texas Children's Provides New Breakthrough Treatment for Patient with Rare Neurological Disorder

HOUSTON, Aug. 19, 2025 /PRNewswire/ -- Texas Children's is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency.

Aromatic l-amino acid decarboxylase (AADC) deficiency is an extremely rare, inherited neurological disorder that prevents the brain from producing dopamine and serotonin — essential chemicals for controlling movement, mood and basic nervous system functions. The literature reports approximately 350 people with this condition worldwide. Historically, there was no cure or approved treatment for AADC, and the shortened life expectancy was estimated between five and seven years of age.

"Texas Children's was the largest contributor to the clinical trial in the U.S. that led to this drug's approval," said Dr. Daniel J. Curry, who performed the six-hour surgery. Dr. Curry is Director of Functional Neurosurgery and Epilepsy Surgery at Texas Children's Hospital and Professor of Neurological Surgery at Baylor College of Medicine. "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. This is the first step in hopefully many future strides toward the molecular correction of inborn deficits for which there used to be no cure."

Kebilidi (eladocagene exuparvovec-tneq) was approved on November 13, 2024, after a clinical trial involving Texas Children's Hospital and two other testing sites. It is a modified adeno-associated virus serotype 2 (AAV2) vector-based gene therapy administered by a neurosurgeon directly into the brain's putamen area, which is involved in learning and motor control. The procedure is a minimally invasive stereotactic neurosurgery that includes four infusions during one surgical session. 

The Texas Children's patient who received the treatment, a 3-year-old girl, first showed signs of developmental delay when she was 7 months old. She had poor muscle control and difficulty holding up her head — one of the common symptoms of AADC deficiency, along with muscle weakness, movement disorders and seizures. Genetic testing confirmed her diagnosis when she was 18 months old, and her local care team has managed her condition with medication and physical therapy until this breakthrough treatment at Texas Children's became available.

"The treatment was successfully administered with no complications," said Dr. Curry. "After a two-week stay in the hospital for follow-up care and monitoring, she and her family are now back home and feeling very optimistic. The therapy is expected to take 2-6 months to show measurable impact, but she is already showing signs of increased energy and happiness."

Dr. Curry helped develop the Texas Children's Intracerebral Gene Therapy Program with investigations into intraputamenal gene therapy for AADC deficiency, as well as investigations into gene therapy for Rett syndrome and NGLY-1 deficiency. Dr. Curry is also Director of the Neuroinfusion Service at Texas Children's, conducting intracerebral enzyme replacement therapy in Batten's disease and other neurodegenerative diseases.

"With these exciting advancements in gene delivery and surgical precision, we can not only treat the root cause of diseases in the brain, but hopefully reverse them," said Dr. Curry. "This will make a life-changing difference for so many patients." 

Learn more about Texas Children's Pediatric Neurosurgery & Neurosurgeons.

About Texas Children's

Texas Children's, a nonprofit health care organization, is committed to creating a healthier future for children and women throughout the global community by leading in patient care, education and research. Consistently ranked as the best children's hospital in Texas and among the top in the nation, Texas Children's has garnered widespread recognition for its expertise and breakthroughs in pediatric and women's health. The system includes the Texas Children's Duncan NRI; the Feigin Tower for pediatric research; Texas Children's Pavilion for Women, a comprehensive obstetrics/gynecology facility focusing on high-risk births; Texas Children's Hospital West Campus, a community hospital in suburban West Houston; Texas Children's Hospital The Woodlands, the first hospital devoted to children's care for communities north of Houston and Texas Children's Hospital North Austin, the new state-of-the-art facility providing world-class pediatric and maternal care to Austin families. The organization also created Texas Children's Health Plan, the nation's first HMO focused on children; Texas Children's Pediatrics, the largest pediatric primary care network in the country; Texas Children's Urgent Care clinics that specialize in after-hours care tailored specifically for children; and a global health program that is channeling care to children and women all over the world. Texas Children's Hospital is affiliated with Baylor College of Medicine. For more information, visit www.texaschildrens.org. 

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SOURCE Texas Children's Hospital