PTC-AADC Infusion Into Brain Shows Promise Against Symptoms

PTC-AADC Infusion Into Brain Shows Promise Against Symptoms

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PTC-AADC Infusion Into Brain Shows Promise Against Symptoms

Delivering the investigational gene therapy PTC-AADC to a specific part of the brain, called the putamen, significantly and effectively lessens motor and non-motor symptoms in children with aromatic L-amino acid decarboxylase (AADC) deficiency, a review-like study highlights. “Before this treatment, the children with AADC deficiency couldn’t lift their heads, but now some can sit and stand with help, and have even begun learning to talk,” Paul Wuh-Liang Hwu, MD, PhD, of the National Taiwan University…

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