Texas Children’s provides new breakthrough treatment for patient with AADC deficiency

Texas Children’s neurosurgeon Dr. Daniel Curry leads first-ever FDA-approved gene therapy treatment for AADC deficiency. ©Texas Children’s Hospital

On August 19, 2025, Texas Children’s, a non-profit healthcare organization, has announced that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for Aromatic l-amino acid decarboxylase (AADC) deficiency, Kebilidi® (eladocagene exuparvovec-tneq). This extremely rare, inherited neurological disorder 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

A three-year-old patient
Kebilidi®, an AAV2 gene therapy containing the cDNA of the human Dopa-DecarboxylaseDDC gene (AAV2-hAADC), administered by a neurosurgeon directly into the brain’s putamen area. The procedure is a minimally invasive stereotactic neurosurgery that includes four infusions during one surgical session. This gene therapy has been developed by PTC Therapeutics and was approved on November 13, 2024, after a clinical trial involving Texas Children’s Hospital and two other testing sites. 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.”

20/08/2025