PTC-AADC Gene Therapy Safe, Showing Promise for Children

PTC-AADC Gene Therapy Safe, Showing Promise for Children

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PTC-AADC Gene Therapy Safe, Showing Promise for Children

Using PTC-AADC gene therapy in children with aromatic l-amino acid decarboxylase (AADC) deficiency was found to be relatively safe and led to improvements in motor function, cognitive abilities, and breathing, a review of six clinical trials found. The therapy also showed benefits in feeding, mood, and sleep among boys and girls with the genetic and neurometabolic disease. The review study, “Gene Therapy in Movement Disorders: A Systematic Review of Ongoing and Completed Clinical Trials,” was…

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