UK Delivers First NHS-Commissioned Brain Gene Therapy to Youngest Patient

Published on Independent UK / LinkedIn via Spencer Knight

A three-year-old girl with a rare disorder called AADC deficiency has become the youngest patient to receive brain-directed gene therapy under the UK’s NHS system. The treatment, developed by PTC Therapeutics and delivered at Great Ormond Street Hospital, infused a modified virus directly into her brain to restore dopamine production. Just months after receiving the therapy, the patient began smiling, reaching, holding her head up, and rolling over, milestones once considered impossible for her condition.

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👉 Girl, 3, becomes youngest patient to receive groundbreaking treatment

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